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Posts Tagged ‘medical’

Helpful Resources and Forms for Pet Owners | Animal Medical Center of …

Tuesday, June 3rd, 2025

These resources can offer additional information for pet owners.

The American Veterinary Medical Association (AVMA) offers a wealth of reputable resources and tips for pet care on its website. They provide guidance about various aspects of pet care, including wellness, behavior, and safety. Visit the AVMA's pet owners section for a comprehensive source of pet care tips and guidelines.

The Association of Reptilian and Amphibian Veterinarians (ARAV) is a respected organization focused on reptile and amphibian health, making it a trustworthy source for reptile owners seeking expert guidance and information. You can visit the ARAVs owner resources to access a variety of articles and guidelines on reptile care, health, husbandry, and species-specific information.

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Helpful Resources and Forms for Pet Owners | Animal Medical Center of ...

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Context Therapeutics Announces Chief Medical Officer Transition

Tuesday, May 6th, 2025

Board Member Dr. Karen Smith to serve as Interim Chief Medical Officer Board Member Dr. Karen Smith to serve as Interim Chief Medical Officer

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Context Therapeutics Announces Chief Medical Officer Transition

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Ophthalmology Services | Brown University Health

Tuesday, May 6th, 2025

Brown University Health Ophthalmology Services

Brown University Health provides the full spectrum of ophthalmology services, from diagnosis and treatment of common eye diseases to cataract surgery, ophthalmic plastic and reconstructive surgery. Our ophthalmology team sees referrals from within the Brown University Health system as well as new patients.

Our highly skilled ophthalmology providers bring years of experience and deliver comprehensive vision care to the southern New England community. They are nationally recognized physicians, many of whom also teach students and residents of The Warren Alpert Medical School of Brown University and conduct research in the field.

Ophthalmology is the branch of medicine concerning the diagnosis and treatment of disorders of the eye. Ophthalmologists receive advanced training to practice medicine and surgery and to diagnose and treat a wider range of conditions.

An ophthalmologist can diagnose and treat all eye diseases, perform eye surgery and prescribe and fit eyeglasses and contact lenses. Some of the common conditions ophthalmologists diagnose and treat are glaucoma, cataracts, disorders with neurological causes such as double vision or vision loss, retinal issues, corneal issues, and issues that require reconstructive surgery.

Many people see an ophthalmologist when they experience chronic or severe vision symptoms, such as:

Because they are advanced medical providers, many ophthalmologists also take part in scientific research on the causes and cures for eye diseases and vision disorders.

Learn more about ophthalmology services

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Ophthalmology Services | Brown University Health

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Georgetown resident leads Indiana Veterinary Medical Association board – News and Tribune

Saturday, April 5th, 2025

Georgetown resident leads Indiana Veterinary Medical Association board  News and Tribune

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Georgetown resident leads Indiana Veterinary Medical Association board - News and Tribune

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Creative Medical Technology Holdings Expands Collaboration with Greenstone Biosciences to Accelerate iPSCelz – EIN News

Friday, February 7th, 2025

Creative Medical Technology Holdings Expands Collaboration with Greenstone Biosciences to Accelerate iPSCelz  EIN News

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Rheumatoid Arthritis Basics: Overview, Symptoms, and Causes

Monday, January 27th, 2025

Rheumatoid arthritis (RA) is a chronic (long-lasting) disease that mostly affects joints, such as the wrist, hands, feet, spine, knees, and jaw. In joints, RA causes inflammation that leads to:

Rheumatoid arthritis is an autoimmune disorder because the immune system attacks the healthy joint tissues. Normally, the immune system helps protect the body from infection and disease.

RA may cause you to feel unusually tired, to have occasional fevers, and to have a loss of appetite. It also may cause other medical problems in the heart, lungs, blood, nerves, eyes, and skin.

Treatments can help people with the disease to lead productive lives.

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Rheumatoid Arthritis Basics: Overview, Symptoms, and Causes

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Patient Care | Johns Hopkins Department of Genetic Medicine

Monday, January 6th, 2025

More than 2,000 people of all ages from across the globe seek information, diagnoses and ongoing care from experts at the McKusick-Nathans Institute of Genetic Medicine | Department of Genetic Medicine. The departmentis a hub of genetics knowledge and care that has moved medicine forward to bring decades of research advances to benefit human health. This is where the field of medical genetics was born and developed.

Johns Hopkins genetics experts are leaders and pioneers in their fields. They use the worlds most advanced technology, some of which was developed by our own scientific teams, to diagnose genetic conditions.

Our clinical team includes physicians, genetic counselors, dieticians, nurses, nurse practitioners and physician assistants who coordinate to develop accurate and timely diagnoses.

Our clinics specialize in unraveling the signs and symptoms of conditions to hone in on a diagnosis, whether the condition is extremely rare or more common. Your team is made up of the worlds experts in deciphering these clues to conditions and in managing your care.

Whether you are a newborn or in your retirement years, experts at our clinics will follow your care for life. Your lifelong link to experts at Johns Hopkins will help you stay informed on the latest advances in care for your condition. We also coordinate care with the Kennedy Krieger Institute, other medical centers and your local health care providers.

Our team will help you navigate the complex medical care that people with genetic conditions may need. We will make referrals and help to arrange appointments with specialists who have extensive knowledge and experience in treatments for people with genetic diseases. We work to get insurance coverage for genetic testing as well as additional needs, such as physical and speech therapy, and equipment, such as wheelchair and communication devices. We also help parents of students with genetic diseases work witheducators to meet their childs medical and learning needs.

Visit our COVID-19 resources page for information about what you can do to stay healthy.

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Patient Care | Johns Hopkins Department of Genetic Medicine

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Request an Appointment | Johns Hopkins Institute of Genetic Medicine

Monday, January 6th, 2025

To request an appointment in any of our genetics clinics, please call410-955-3071.

If this is your first visit to a genetics clinic at Johns Hopkins, the following steps will help you navigate making an appointment. From start to finish, scheduling an appointment may take up to 10 days, depending on the speed of insurance clearance, receiving records, and other factors.

Call our central appointment line at 410-955-3071. Our staff in the genetics office will walk you through the steps to making an appointment. They will collect general and insurance information about the patient and will send you a medical history questionnaire.

Complete one of the following medical history questionnaires, and fax the completed questionnaire to 410-367-3231.

Your primary care physician can help you complete the questionnaire. Genetic counselors review the questionnaire to determine each persons medical urgency and the appropriate medical providers to schedule the appointment. People with medical urgency who should receive appointments sooner than the general population of our patients include infants under six months of age, children whose physicians diagnosed them as failure to thrive or children who have lost developmental milestones.Generally, our next available appointments are four to six months from the time you first call our appointment line.

Our financial specialists will review your insurance information to confirm that it is active and will cover a visit with a medical geneticist, genetic counselor, dieticianand nurse. They will also help obtain referralsand will determine eligibility and coverage for genetic testing. You can help make this process faster by asking your primary care provider to fax a referral and records to 410-367-3231.

After your questionnaire and insurance status have been reviewed, our scheduling staff will contact you to schedule the first available appointment.

Questions about the status of your appointment?Call the main appointment line at 410-955-3071, Option 1

If you have been seen at one of our genetics clinics within three years, call 410-955-3071, option 2, to schedule your follow-up appointment.

If three or more years have passed since your last appointment at one of our genetics clinics, please follow the instructions for new patients.You will not need to submit a new medical questionnaire. The genetic counselors will review your genetic medical record.

Book follow-up visits early!Available appointments fill quickly, so dont delay in scheduling your next visit.

Johns Hopkins Medicine International pairs you with a medical concierge to arrange all aspects of your medical visit, paying special attention to your personal, cultural and travel-related needs. Your medical concierge can arrange consultations and treatment plans with the most appropriate specialists. Johns Hopkins Medicine International also provides language interpretation, financial counseling, assistance with travel arrangements and anything else to help make Johns Hopkins feel as close to home as possible.

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Request an Appointment | Johns Hopkins Institute of Genetic Medicine

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100 cell and gene therapy leaders to watch in 2025

Saturday, December 28th, 2024

1Bluebird BioSomerville, MA, USAGene therapies for genetic diseasesZynteglo, Skysona, LyfgeniaPublic (BLUE)Multiple approved gene therapies for rare diseases. Patents (20202024): 15 total (10 gene, 1 vector, 4 cancer)2NovartisBasel, SwitzerlandPioneer: CAR-T (Kymriah), Zolgensma (SMA)Kymriah, ZolgensmaPublicRobust pipeline, global CGT leader. Patents (20202024): 95 total (47 gene, 8 vector, 40 cancer)3Gilead Sciences/Kite PharmaFoster City, CA, USACAR-T therapies for oncologyYescarta, TecartusPublic (GILD)Leading CAR-T franchise in oncology. Patents (20202024): 40 total (6 vector, 34 cancer). 2024 sales data strong: Q2: $521M combined, Q3: ~$485M, steady performance.4Bristol Myers SquibbNew York, NY, USAMultiple CAR-T approvalsAbecma, BreyanziPublic (BMY)Deep CAR-T pipeline, oncology focus. Patents (20202024): 164 total (92 gene, 12 vector, 60 cancer). Q3 2024 total revenue +8% YoY to $11.9B; growth portfolio (incl. Breyanzi, Abecma) +18% to $5.8B.5RocheBasel, SwitzerlandInherited retinal disease gene therapyLuxturnaPublicIntegrated CGT pipeline, global presence. Patents (20202024): 61 total (18 gene, 40 vector, 3 cancer). Elevidys sales Q3 2024: ~30M CHF, exceeding analyst expectations.6PfizerNew York, NY, USAGene therapy in hemophilia B, othersBeqvezPublic (PFE)Partnering in gene therapies, robust R&D. Patents (20202024): 25 total (6 gene, 5 vector, 14 cancer)7BioMarin PharmaceuticalSan Rafael, CA, USARare disease gene therapy (hem A)RoctavianPublic (BMRN)Pioneer in rare disease gene therapy. Patents (20202024): 13 total (13 gene)8Vertex PharmaceuticalsBoston, MA, USAExa-cel for SCD/-thalassemiaCasgevy (Exa-cel)Public (VRTX)Strong CF base, gene editing collaborations. Patents (20202024): 19 total (16 gene, 3 vector)9Sarepta TherapeuticsCambridge, MA, USADMD gene therapyElevidysPublic (SRPT)Leader in RNA/gene for neuromuscular disease. Patents (20202024): 11 total (11 gene)10Adaptimmune TherapeuticsAbingdon, UKEngineered T-cells for solid tumorsTecelraPublic (ADAP)SPEAR T-cell therapies. Patents (20202024): 22 total (3 vector, 19 cancer)11Autolus TherapeuticsLondon, UKNext-gen CAR-TAucatzylPublic (AUTL)Modular CAR-T platforms. Patents (20202024): 45 total (5 gene, 40 cancer)12Krystal BiotechPittsburgh, PA, USAGene therapy for DEBVyjuvekPublic (KRYS)Topical gene therapy for rare skin disorders. Patents (20202024): 9 total (9 gene)13Orchard TherapeuticsLondon, UKEx vivo gene therapies (rare)LenmeldyPublic (ORTX)Lentiviral ex vivo for inherited disorders14Gamida CellBoston, MA, USACell therapy for BMT (Omisirge)OmisirgePublic (GMDA)Improving bone marrow transplant outcomes15EnzyvantDurham, NC, USAAllogeneic processed thymus tissueRethymicPrivate/SubsidiaryThymus-based regenerative medicine16MesoblastMelbourne, AustraliaAllogeneic MSC therapy (GvHD)RyoncilPublic (MESO)MSC platform for inflammatory diseases17Astellas Gene TherapiesBoston, MA, USANeuromuscular & ocular gene therapiesNonePublic (ALPMY)Expanding CNS/ocular pipeline. Patents (20202024): 19 total (14 gene, 5 cancer)18BayerLeverkusen, GermanyHemophilia gene therapy pipelineNonePublicInvested heavily in CGT (AskBio, BlueRock integrated). Patents (20202024): 12 total (11 gene, 1 vector)19Intellia TherapeuticsCambridge, MA, USACRISPR in vivo editingNonePublic (NTLA)First systemic in vivo CRISPR trial. Patents (20202024): 17 total (14 gene, 3 vector)20Beam TherapeuticsCambridge, MA, USABase editing gene therapiesNonePublic (BEAM)Base editing precision. Patents (20202024): 11 total (1 gene, 10 vector)21REGENXBIORockville, MD, USAAAV vector platformNonePublic (RGNX)NAV AAV vectors licensed broadly. Patents (20202024): 10 total (8 gene)22uniQureLexington, MA, USAAAV gene therapiesHemgenix (with CSL Behring)Public (QURE)Hemophilia B gene therapy23Freeline TherapeuticsStevenage, UKAAV for hemophilia & FabryNonePublic (FRLN)Next-gen AAV capsids, metabolic/bleeding disorders24AvrobioCambridge, MA, USALentiviral gene therapies for lysosomal disordersNonePublic (AVRO)Ex vivo lentiviral for metabolic diseases. Funding Status: M&A, Last Funding Date: Feb 1, 2018, $60,000,000 (Series B), Total Funding Amount: $85,000,000, Top Investors: Cormorant Asset Management, Citadel, Atlas Venture, SV Health Investors, Leerink Partners25Rocket PharmaceuticalsNew York, NY, USALentiviral & AAV for rare diseasesNonePublic (RCKT)Hematologic and cardiac gene therapy pipeline26Passage BioPhiladelphia, PA, USACNS AAV gene therapiesNonePublic (PASG)UPenn partnership, rare CNS disorders27Solid BiosciencesCambridge, MA, USADMD gene therapyNonePublic (SLDB)Microdystrophin gene therapy for DMD28Homology MedicinesBedford, MA, USAIn vivo gene editing & gene therapyNonePublic (FIXX)AAVHSC platform for gene correction. Patents (20202024): 8 total (8 gene)29ElevateBioWaltham, MA, USACGT innovation centerNonePrivate ($401M Series D)Enabling platform for multiple CGTs. Funding Status: Late Stage Venture, Last Funding Date: May 24, 2023, Last Funding Amount: $401,000,000 (Series D), Total Funding Amount: $1,246,000,000, Top Investors: Novo Nordisk, Fidelity, Vertex Ventures, Invus, Surveyor Capital30Sana BiotechnologySeattle, WA, USAEx/in vivo CGTNonePublic (SANA)Fusogenix, hypoimmune platforms31Fate TherapeuticsSan Diego, CA, USAiPSC immunotherapiesNonePublic (FATE)Off-the-shelf NK & T-cell therapies. Patents (20202024): 7 total (7 cancer)32Poseida TherapeuticsSan Diego, CA, USAGene editing allogeneic CAR-TNonePublic (PSTX)* Roche is acquiringNon-viral gene editing (piggyBac). Patents (20202024): 20 total (11 gene, 3 vector, 6 cancer)33Caribou BiosciencesBerkeley, CA, USACRISPR-edited cell therapiesNonePublic (CRBU)chRDNAs for precision CRISPR editing. Patents (20202024): 48 total (4 gene, 33 vector, 11 cancer)34CellectisParis, FranceTALEN-based allogeneic CAR-TNonePublic (CLLS)TALEN gene editing, off-the-shelf CAR-T. Patents (20202024): 91 total (12 gene, 20 vector, 59 cancer)35MeiraGTxLondon, UKAAV for ocular & neuroNonePublic (MGTX)Vertically integrated gene therapy company. Patents (20202024): 15 total (10 vector, 5 gene*). Additional Funding Info (for Tmunity Therapeutics Rank #35): M&A, Last Funding Date: Oct 31, 2019, $75,000,000 (Series B), Total Funding Amount: $220,000,000, Top Investors: Gilead Sciences, Andreessen Horowitz, University of Pennsylvania, Kleiner Perkins, Lilly Asia Ventures364D Molecular TherapeuticsEmeryville, CA, USAEngineered AAV vectorsNonePublic (FDMT)Custom AAV capsids for multiple indications. Patents (20202024): 34 total (34 gene)37Abeona TherapeuticsNew York, NY, USARare disease gene therapiesNonePublic (ABEO)Focus on RDEB and CLN138Adverum BiotechnologiesRedwood City, CA, USAAAV ocular gene therapyNonePublic (ADVM)Wet AMD gene therapy. Patents (20202024): 26 total (23 gene, 3 vector)39Generation BioCambridge, MA, USANon-viral genetic medicinesNonePublic (GBIO)Closed-ended DNA platform40Dyno TherapeuticsCambridge, MA, USAAI-driven AAV engineeringNonePrivateAI to optimize AAV capsids41MetagenomiEmeryville, CA, USANovel CRISPR systemsNonePublic (MGX)Metagenomic enzyme discovery for gene editing. Patents (20202024): 11 total (1 gene, 10 vector)42EdiGeneBeijing, ChinaGene editing & therapyNonePrivateCRISPR-based therapies in development. Patents (20202024): 6 total (6 vector)43CelularityFlorham Park, NJ, USAPlacental-derived cell therapiesNonePublic (CELU)Off-the-shelf NK cell therapies44Rubius TherapeuticsCambridge, MA, USARed Cell TherapeuticsNonePublic (RUBY)Engineering RBCs for immuno-oncology. Patents (20202024): 8 total (7 gene, 1 cancer)45ArcellxGermantown, MD, USAControllable CAR-TNonePublic (ACLX)ARC-sparX platform. Patents (20202024): 5 total (5 cancer)46AstraveusParis, FranceCGT manufacturing solutions (with innovation)NoneSeed 10.4MMicrofluidic platform for scalable manufacturing. Funding Status: Seed, Last Funding Date: Oct 24, 2023, 10,400,000 (Grant), Total Funding Amount: 28,872,000, Top Investors: Bpifrance, EASME EU Executive Agency for SMEs, Johnson & Johnson Innovation JJDC, Bpifrance Large Venture, M Ventures47Inceptor BioRaleigh, NC, USACell therapy for tough cancersNoneEarly Stage VentureDiversified cell therapy pipeline. Funding Status: Early Stage Venture, Last Funding Date: Oct 20, 2022, $15,875,000 (Debt Financing), Total Funding Amount: $87,645,000, Top Investors: Kineticos Ventures, Kineticos Disruptor Fund48Tevogen BioWarren, NJ, USAOff-the-shelf T-cell therapiesNonePublic (TVGN)Allogeneic T-cells for viral infections/cancer. Funding Status: IPO, Last Funding Date: May 10, 2024, $36,000,000 (Post-IPO Debt), Total Funding Amount: $58,000,000, Top Investors: HMP Partners49CSL BehringMelbourne, AustraliaGlobal biotech in immunology/heme/CGTNonePublic (CSL)Hemophilia B gene therapy & plasma products50CRISPR TherapeuticsZug, Switzerland & Boston, MACRISPR/Cas9 gene editing therapiesExa-cel (with Vertex)Public (CRSP)Pipeline in hemoglobinopathies, oncology. Patents (20202024): 57 total (46 gene, 6 vector, 5 cancer)51ImmunoACTMumbai, IndiaAdvanced CGT startupNone800M CorporateCAR-T development for emerging markets. Funding Status: Corporate Round (Unlisted as IPO/M&A), Last Funding Date: May 31, 2023, 800,000,000, Total Funding Amount: 1,397,500,000, Top Investors: Laurus Labs52Coave TherapeuticsParis, FranceGene therapies for ocular/CNSNonePrivate (33.1M Series B)AAV-ligand conjugates53Celyad OncologyBelgiumAllogeneic CAR-T for oncologyNonePublic (CYAD)shRNA-based allogeneic CAR-T54Chimeron BioPhiladelphia, PA, USASelf-amplifying RNA therapeuticsNone$4.33M fundingChaESAR RNA platform55Deep GenomicsToronto, CanadaAI-driven RNA therapeuticsNone$241M totalBigRNA AI for RNA drug design56DendreonSeal Beach, CA, USACellular immunotherapy for prostate cancerPROVENGECommercial revenueAutologous cell therapy pioneer57Editas MedicineCambridge, MA, USACRISPR-based gene editing therapiesNone~$931.6M raisedIn vivo & ex vivo CRISPR platform. Patents (20202024): 48 total (21 gene, 26 vector, 1 cancer)58eGenesisCambridge, MA, USAGene-edited xenotransplantationNone$191M Series DMulti-gene-edited porcine organs59EyevensysParis, FranceNon-viral gene therapy for eye diseasesNone$44.1M fundingElectroporation-based ocular gene delivery60Ferring PharmaceuticalsSaint-Prex, SwitzerlandADSTILADRIN for bladder cancerADSTILADRINPrivate; $500M royalty dealGene therapy in oncology61Forte BiosciencesDallas, TX, USAAnti-CD122 mAb for autoimmuneNone$53M private placementTargeting IL-2 pathway disorders62GenascencePalo Alto, CA, USAGene therapy for osteoarthritisNoneFDA Fast Track GNSC-001IL-1Ra gene therapy for OA63GenSight BiologicsParis, FranceGene therapies for retinal diseasesNonePublic (SIGHT.PA)Mitochondrial targeting, optogenetics64Cabaletta BioPhiladelphia, PA, USAT-cell therapies for autoimmune diseaseNonePublic (CABA)CAART for B-cell-mediated conditions65Capricor TherapeuticsSan Diego, CA, USACell & exosome-based therapies (DMD)NonePublic (CAPR)StealthX exosome platform66RegeneronTarrytown, NY, USAExpanding in CGT, advanced biologicsNone (CGT)Public (REGN)Acquired cell therapy programs, DB-OTO gene therapy67RiboKunshan, ChinaRNAi therapeuticsNoneMultiple RMB roundsGalNAc RNAi for liver/CV/metabolic68TakedaTokyo, JapanGlobal pharma investing in CGTNonePublic (TAK)Cell therapy facility, gene therapy collaborations69Ultragenyx PharmaceuticalNovato, CA, USARare & ultra-rare diseases (gene therapy)NonePublic (RARE)Diverse biologics/gene therapy pipeline70VinetiSan Francisco, CA, USAPersonalized therapy management platformNoneSeries A-C fundingSoftware for CGT supply chain71VericelCambridge, MA, USACell therapies for cartilage & burnsMACI, Epicel, NexoBridPublic (VCEL)Strong revenue, expanding indications72Verve TherapeuticsBoston, MA, USAGene editing for cardiovascular diseaseNonePublic (VERV)Base editing for LDL-C/triglycerides73Vivet TherapeuticsParis, FranceGene therapies for metabolic diseasesNonePrivateVTX-801 for Wilson disease (Phase 1/2)74Voyager TherapeuticsCambridge, MA, USAGene therapies for neurological diseasesNonePublic (VYGR)TRACER capsids, partnered CNS programs75Sangamo TherapeuticsBrisbane, CA, USAZinc finger & AAV gene therapiesNonePublic (SGMO)ZFN platform, Fabry, hemophilia programs76SQZ BiotechnologiesWatertown, MA, USACell therapy for HPV16+ tumorsNonePublic (SQZ)AAC & eAPC platforms, strategic restructuring77Arrowhead PharmaceuticalsPasadena, CA, USARNAi therapeutics (cardiometabolic & more)NonePublic (ARWR)SiRNA pipeline, strong partnerships78Alnylam PharmaceuticalsCambridge, MA, USARNAi therapies for various diseasesOnpattro, Givlaari, Oxlumo, AmvuttraPublic (ALNY)RNAi pioneer with multiple approved products. Patents (20202024): 73 total (73 gene)79Aspen NeuroscienceSan Diego, CA, USAAutologous iPSC for Parkinsons diseaseNonePrivate (> $220M raised)iPSC-based personalized cell therapy80American Gene Technologies (AGT)Rockville, MD, USAGene therapy for HIV & rare diseasesNone~$78M fundingHIV functional cure in Phase 181Ionis PharmaceuticalsCarlsbad, CA, USAAntisense therapiesSpinraza, Tegsedi, WaylivraPublic (IONS)Antisense leader, broad pipeline. Patents (20202024): 62 total (60 gene, 2 vector)82Allogene TherapeuticsSouth San Francisco, CA, USAAllogeneic CAR T therapiesNonePublic (ALLO)Off-the-shelf CAR T for hematologic & solid tumors83NexImmuneGaithersburg, MD, USAAIM nanoparticle immunotherapyNonePublic (NEXI)Artificial Immune Modulation platform84NextCureBeltsville, MD, USAImmunomedicines for oncologyNonePublic (NXTC)B7-H4 ADC, immunotherapy pipeline85Oxford BiomedicaOxford, UKVector platform (Lentiviral), integrated innovationNonePublic (LSE:OXB)Leading lentiviral vector developer & collaborator86Pluri Inc.Haifa, Israel3D cell-based technology across industriesNonePublic (PLUR)3D cell expansion platform87ReNeuronBridgend, UKExosome-based therapeuticsNonePublic (RENE.L)CustomEX platform for targeted delivery88Arcturus TherapeuticsSan Diego, CA, USAmRNA medicines & vaccinesNonePublic (ARCT)Self-amplifying mRNA vaccines/therapies. Patents (20202024): 23 total (23 gene)89Silence TherapeuticsLondon, UKRNAi therapeutics targeting liver genesNonePublic (SLN)GalNAc-siRNA pipeline90BioNTechMainz, GermanymRNA-based immunotherapiesComirnaty (COVID-19 vaccine)Public (BNTX)mRNA cancer immunotherapies, global leader. Patents (20202024): 36 total (29 gene, 7 vector)91ModernaCambridge, MA, USAmRNA therapeutics & vaccinesSpikevax (COVID-19 vaccine)Public (MRNA)Expanding mRNA platform to rare diseases. Patents (20202024): 140 total (124 gene, 16 vector)92CureVacTbingen, GermanymRNA therapeutics & vaccinesNonePublic (CVAC)mRNA platform for prophylactic & therapeutic use. Patents (20202024): 65 total (56 gene, 9 vector)93Inovio PharmaceuticalsPlymouth Meeting, PA, USADNA medicines (cancer, infectious diseases)NonePublic (INO)Electroporation delivery of DNA plasmids. Patents (20202024): 28 total (25 gene, 3 vector)94Senti BiosciencesSouth San Francisco, CA, USAGene circuit-engineered cell & gene therapiesNonePublic (SNTI)Programmable gene circuits for cell therapies95Graphite BioSouth San Francisco, CA, USACRISPR gene editing therapiesNonePublic (GRPH)Precise gene correction for genetic diseases96Century TherapeuticsPhiladelphia, PA, USAAllogeneic iPSC-derived cell therapiesNonePublic (IPSC)iPSC platform for NK & T-cell therapies97ArsenalBioSan Francisco, CA, USAProgrammable cell therapies for cancerNonePrivateEngineered T-cells with synthetic biology98Homestead BioPharmaHouston, TX, USAGene therapies targeting AML & cancersNonePrivateEarly-stage gene therapy for oncology99Anew MedicalOmaha, NE, USACGT for cancer/neurologicalNoneIPO $15MDeveloping gene/cell therapies for CNS & cancer. Funding Status: IPO, Last Funding Date: May 26, 2023, $15,000,000 (Post-IPO Debt), Total Funding Amount: $15,000,000, Top Investors: Gaensel Energy Group100AmbuleroMiami, FL, USAVascular gene therapiesNoneSeed $5.5MGene therapies for vascular/ischemic diseases. Funding Status: Seed, Last Funding Date: Jan 25, 2021, $5,500,000 (Seed), Total Funding Amount: $5,500,000, Top Investors: Orphinic Scientific

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100 cell and gene therapy leaders to watch in 2025

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Nebraska Medicine administers novel gene therapy to first hemophilia …

Saturday, December 28th, 2024

Julie AndersonOmaha World-Herald

After more than four decades of infusing himself with the blood clotting factor his body cant make, Chad Stevens decided it was time to try something new.

Stevens, 63, suffers from hemophilia B, a bleeding disorder caused by a genetic mutation that affects production of a type of protein known as factor 9. Over the years, bleeds have damaged his joints. His ankles have been fused, his knees and elbows have severe damage. And successfully hitting a vein to infuse himself as he got older wasnt getting any easier.

In mid-October, Stevens traveled from his home town of Newdale, Idaho, to Omahas Nebraska Medical Center, where he became the hospitals first patient to receive the first gene therapy approved for his condition.

Called Hemgenix, the therapy doesnt fix the damaged gene. Instead, a modified virus delivers the working gene to the liver, providing the instructions his body needs to make the factor on its own. The medical center is the first hospital in the region to become an administration site for the therapy, according to drug-maker CSL Behring.

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Since then, Stevens hasnt had any bleeds or needed to infuse clotting factor. He said he hopes the therapy will provide enough to take him from severe hemophilia to a milder version that might require infusions only for a severe trauma or surgery.

Thats really promising, Stevens said. I hate to get too excited about it, because you never know whats going to happen. But Im quite thrilled with it.

So is Dr. Alex Nester, a hematologist with Nebraska Medicine who specializes in benign or non-cancerous blood conditions, including bleeding disorders and sickle cell disease.

Its incredible, he said. Its (been) a dream in the hemophilia community for 20-plus years.

The treatment, approved by the Food and Drug Administration in 2022, is one of a number of gene therapies that have trickled out in recent years for a variety of genetic conditions. The FDA approved a separate gene therapy for hemophilia A last year. The agency also has approved two gene therapies for sickle cell disease, another inherited blood disorder that causes red blood cells to become misshapen, block blood flow and cause painful episodes.

Kim Phelan, CEO of The Coalition for Hemophilia B, said the lasting advantages of the gene therapy include reduced joint damage, fewer hospitalizations and a better quality of life for people with hemophilia.

An estimated 7,000 people in the U.S. have hemophilia B, and approximately 17,000 have hemophilia A, which involves a different blood clotting factor.

After more than 25 years of anticipation and hope, individuals with hemophilia now have access to a groundbreaking therapy that offers the potential for greater independence and a more normalized life, she said.

Gene therapy at Nebraska Medicine

At Nebraska Medicine, the addition of the gene therapy builds on the work of the team involved in bone marrow transplants and cellular therapies, including CAR-T, or chimeric antigen receptor T-cell therapy. That treatment involves removing patients immune cells from their bodies and genetically engineering them to recognize and attack their cancer.

Dr. Matthew Lunning, medical director of gene and cellular therapy at Nebraska Medicine, said earlier this fall that the team has used CAR-T to treat hundreds of lymphoma and leukemia patients since the late 2010s.

Earlier this year, he and his team used CAR-T for the first time to treat an Omaha woman with lupus, an autoimmune disease, as part of a multi-site clinical trial. He credited Nebraska Medicines leaders for making the investment required to offer such ground-breaking therapies.

Still, gene therapies, according to news reports, have been somewhat slow to catch on. In the case of hemophilia, Nester said he suspects that may be a result of the complex modern history of the condition.

By the 1980s, he said, hemophilia patients who suffered trauma were given a concentrated form of the missing proteins when they needed help getting their blood to clot. But many contracted infections such as HIV and hepatitis C from contaminated blood products, which killed thousands of those with severe disease. Later, the products were purified but still were reserved for cases of active bleeding. As a result, older patients like Stevens suffered significant joint damage.

In the 1990s, researchers began producing a recombinant version of the missing proteins in hamster cells, similar to the way insulin is made. Children diagnosed with hemophilia could dose themselves with clotting factors to prevent bleeds, he said. That resulted in a generation with no bleeding episodes for years at a time and without the joint damage suffered by older patients.

You dont need a lot of these factors to live a pretty normal life, said Nester, also an assistant professor of medicine in UNMCs oncology and hematology division.

That also means younger patients may have less interest for now in a more permanent solution, he said. Some also may be holding off for newer versions of the gene therapy that are in the pipeline.

Stevens said his parents, on the other hand, were told he probably wouldnt survive his teens. Between his mother and her three sisters, three had children with hemophilia, a total of seven. He was the youngest. He is now the sole survivor. Several died from bleeds and a couple died of complications of AIDs due to the contaminated clotting factor relied on at the time.

It took a big toll on the hemophilia community, he said. It just decimated it, really. So us older ones are pretty lucky to have survived all of that.

Issues with earlier blood products, however, also have made older patients skeptical about new treatments. We like to wait and see how the products are doing out there before you jump on it, he said.

Cost of treatment can run into the millions

Patients also have to weigh the cost. The price for the one-time treatment reportedly was set at $3.5 million.

A spokesperson for CSL Behring said the company has seen an acceleration in the number of people being infused with the therapy since its approval, which the company attributes to its outreach to patients and work with insurers. Some 90% now cover the therapy, and the company also offers a program to help patients with copays. She declined to say, however, how many patients have received the therapy.

But Nester said clotting factors also are costly. Depending on the patient, the source of their factor and their insurance, it may run a half a million dollars a year to keep nothing from happening, he said.

Meanwhile, he said, researchers have seen that the majority of patients who have received the gene therapy are making 10% or more of the normal levels of the missing clotting factor even five years after being treated. That means their bodies are producing at least the preventative dose.

Patients still may have a bleeding episode after twisting their ankle or maybe needing a dose before surgery, Nester said, but, generally speaking, spontaneous bleeds or bleeds associated with minor trauma are gone.

Not every hemophilia patient will qualify for the treatment, however, he said. Patients cant have antibodies to either the virus or the factor theyre missing.

Stevens said his infusions probably cost closer to three-quarters of a million dollars a year. So far, the cost of his gene therapy has been covered. Previously employed in banking in Boise, he retired and applied for Social Security disability benefits on the advice of his doctor after his pain and mobility issues had made it nearly impossible for him to get out of his chair at work.

He moved back to Newdale, population 325, in eastern Idaho. But he didnt like being on disability, because he wasnt giving back. He was elected to the City Council and appointed mayor, a post he continues to hold.

It was just a pleasure to be contributing again, Stevens said.

Since receiving the therapy, he said, he seems to be moving a little better, and his knee isnt bothering him as much. Since the damage was done at an earlier age, he doesnt think the therapy will do much to repair it.

But if we can keep it from getting any worse, Stevens said, thats the goal.

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Comparing Genetics and Molecular Genetics: What’s the Difference?

Thursday, December 19th, 2024

When it comes to the study of genes, there are two different approaches that scientists use to gain a deeper understanding of genetic makeup and its role in various biological processes. These approaches are genetics and molecular genetics. While both involve the analysis and research of genetic material, there are significant differences between the two.

Genetics, as a field of study, focuses on the inheritance and variation of genes in organisms. It involves the examination of traits, such as eye color or height, and the mapping of these traits to specific genes. This approach involves studying the DNA sequences and chromosomes to understand how genes are passed down from one generation to another.

On the other hand, molecular genetics takes a more detailed and intricate look at genes and their functions. It delves into the molecular mechanisms behind genetic processes, such as DNA replication, transcription, and translation. Molecular genetics employs advanced techniques and tools to analyze the structure and function of genes at the molecular level, including the study of specific gene mutations that may cause diseases.

Therefore, the main difference between genetics and molecular genetics lies in their scope and depth of analysis. While genetics looks at the broader picture of gene inheritance and variation, molecular genetics zooms in to elucidate the complex molecular processes and interactions that occur within genes. Both disciplines are valuable in their own right, complementing each other to provide a comprehensive understanding of how genes function and contribute to the diversity of life on Earth.

In the study of genetics, two main approaches are often compared: genetics and molecular genetics. Although these terms may sound similar, they represent different fields of analysis and study.

Genetics is the branch of biology that focuses on the study of genes, heredity, and variation in living organisms. It involves the analysis of inherited traits and the passing of genetic information from one generation to the next. Geneticists use various techniques, such as pedigree analysis and population genetics, to understand how genes are inherited and how they contribute to the diversity of organisms.

Molecular genetics, on the other hand, takes a more detailed and specific approach. It focuses on the study of the structure, function, and organization of genes at a molecular level. Molecular geneticists use advanced techniques, such as DNA sequencing and gene cloning, to analyze the DNA molecules and understand how specific genes function and interact. They study the mechanisms of gene expression, regulation, and mutations, and how they relate to genetic disorders and diseases.

While genetics provides a broad overview of inherited traits and genetic patterns in populations, molecular genetics delves deeper into the molecular mechanisms that underlie these patterns. It is a more specialized field that allows for a more detailed understanding of how genes function and how they contribute to the diversity of life forms.

In summary, genetics and molecular genetics are two distinct but related fields of study. Genetics provides a broader analysis of inherited traits and genetic patterns, while molecular genetics takes a more specific approach in understanding the molecular mechanisms of genes. Both fields are crucial for advancing our knowledge of genetics and its impact on living organisms.

In the field of genetics, the study of inheritance and variation in living organisms has been a subject of fascination since ancient times. However, it wasnt until the mid-20th century that the field took a major leap forward with the advent of molecular genetics.

Prior to the emergence of molecular genetics, the study of genetics primarily focused on observing and analyzing the hereditary traits of organisms through methods such as breeding experiments, statistical analysis, and observation of visible characteristics. This approach, known as classical genetics, provided valuable insights into the patterns of inheritance but had limitations in its ability to interrogate the underlying molecular mechanisms.

Molecular genetics, on the other hand, revolutionized the field by introducing a more detailed and comprehensive approach to the study of genetics. This approach involved the exploration of the structure and function of genes at the molecular level, analyzing the role of DNA and RNA in gene expression, and understanding the mechanisms of mutation and genetic variation.

The comparison between classical genetics and molecular genetics reveals significant differences in their research methodology and analysis. Classical genetics relied on observational data and statistical analysis to infer patterns of inheritance, while molecular genetics employs sophisticated laboratory techniques to manipulate and analyze DNA and RNA molecules.

Furthermore, the advent of molecular genetics has allowed researchers to delve deeper into the intricate mechanisms of genetic inheritance and variation. By studying molecular processes such as DNA replication, transcription, and translation, scientists have gained a more nuanced understanding of how genes interact and contribute to the development and functioning of organisms.

Overall, the emergence of molecular genetics as a distinct discipline has greatly expanded our knowledge of genetics and opened up new avenues of research. Its focus on the molecular level has provided invaluable insights into the complexities of genetic processes and has paved the way for advancements in fields such as biotechnology, genomics, and personalized medicine.

When it comes to the study of genetics and molecular genetics, there are several key concepts to understand. A comparison between genetics and molecular genetics provides insights into the differences in their approaches and analysis.

Genetics is the study of genes and heredity. It focuses on the inheritance of traits from one generation to another. Geneticists analyze the variations and similarities in genes to understand how certain traits are passed on and expressed through generations. They study the genes at a macroscopic level, observing the patterns of inheritance and the effects of genetic mutations on individuals.

Molecular genetics, on the other hand, takes a more microscopic approach. It zooms in on the molecular level to understand the structure and function of genes. By analyzing DNA and RNA sequences, molecular geneticists can unravel the intricacies of genetic information. They study the changes and interactions within genes and delve deeper into the mechanisms of gene expression and regulation.

The main difference between genetics and molecular genetics lies in their level of analysis. Genetics takes a broader approach, while molecular genetics focuses on a more detailed examination of genes and their molecular components. Both fields contribute to the understanding of how genetic information is inherited and expressed, but they employ different methodologies and perspectives.

In conclusion, genetics and molecular genetics are two related fields that study genes and heredity. Genetics focuses on the inheritance of traits at a macroscopic level, while molecular genetics delves into the molecular components and mechanisms of gene expression. By understanding the differences between these two approaches, scientists can gain a more comprehensive understanding of the complexities of genetic information.

In the research field of genetics, the methodology often involves the study of genetic variations and heredity patterns in living organisms. This can be achieved through various techniques such as pedigree analysis, gene mapping, and DNA sequencing. The primary focus of genetic research is to understand the differences and similarities between individuals and populations in terms of their genetic makeup.

On the other hand, molecular genetics is a more specific branch of genetics that focuses on the analysis of DNA and RNA molecules. It involves studying the structure, function, and regulation of genes at a molecular level. Molecular geneticists use techniques like PCR (polymerase chain reaction), gel electrophoresis, and DNA cloning to isolate and analyze specific genes or DNA sequences.

One key difference between genetic research and molecular genetics is the scale at which they operate. While traditional genetics looks at broader genetic traits and inheritance patterns, molecular genetics examines the specific molecular mechanisms behind these traits. This enables researchers to gain a more in-depth understanding of the biological processes involved.

Another difference is the level of detail in the analysis. Genetic research often involves observations at the organism level, such as comparing traits between individuals or populations. In contrast, molecular genetics focuses on the molecular level, analyzing DNA sequences and gene expression patterns.

In summary, the comparison between genetics and molecular genetics reveals the differences in the scope and approach of the two fields. While genetics provides a broader perspective on heredity and genetic variation, molecular genetics delves into the intricate molecular mechanisms underlying these phenomena. Both disciplines contribute valuable insights to our understanding of the genetic basis of life.

Both genetics and molecular genetics have important applications in the field of medicine. While there are some differences in their approaches and methodologies, they both contribute to our understanding of genetic diseases and provide valuable insights for diagnosis, treatment, and prevention.

Genetics is the study of heredity and the variation of inherited traits in organisms. It focuses on the examination of genes, their structures, functions, and how they are transmitted from one generation to another. Genetic analysis has long been used in the medical field to identify the genetic basis of diseases and to assess an individuals risk for developing certain conditions. It has played a crucial role in the identification of genetic disorders, such as Down syndrome and cystic fibrosis. Genetic counseling, which involves assessing an individuals risk for genetic conditions and providing information and support, is another important application of genetics in medicine.

Molecular genetics, on the other hand, takes a more focused approach by studying the structure and function of individual genes at a molecular level. It involves the analysis of DNA, RNA, and proteins to understand how genes are regulated and how they contribute to the development of diseases. The molecular analysis of genes has revolutionized the field of medicine by enabling researchers to identify specific molecular markers for diseases, develop targeted therapies, and predict therapeutic responses. This approach has led to personalized medicine, where treatments can be tailored to an individuals genetic profile.

Both genetics and molecular genetics have contributed significantly to the understanding and treatment of genetic diseases. While genetics provides a broader picture of genetic inheritance and variation, molecular genetics offers a more detailed analysis of genes and their functions. Together, they form a powerful combination for medical research and have paved the way for breakthroughs in the diagnosis, treatment, and prevention of genetic disorders.

When it comes to the field of agriculture, both genetics and molecular genetics play crucial roles in research and advancements. While they may have similarities in their approaches and analyses, there are also significant differences between the two.

Genetics, as a branch of biology, studies the inheritance and variation of traits in living organisms, including plants and animals. In agriculture, genetics is applied to breed selectively and improve the desirable traits of crops and livestock. Through traditional breeding methods, geneticists identify and cross-breed plants and animals with specific desirable traits to create new varieties with enhanced characteristics such as disease resistance, productivity, and quality.

The Role of Molecular Genetics in Agriculture

Molecular genetics takes a more advanced and precise approach compared to traditional genetics. It involves the study of genes and their functions at the molecular level, focusing on analyzing DNA and other genetic materials.

Advancements in molecular genetics have revolutionized the field of agriculture.

While traditional genetics relies on breeding experiments and observations, molecular genetics utilizes various laboratory techniques and technologies to directly manipulate and analyze genetic materials. This includes techniques such as polymerase chain reaction (PCR), gene cloning, and genome sequencing.

Analysis and Study of Genes

Molecular genetics allows for a more in-depth analysis and study of individual genes, their interactions, and their functions. This provides valuable insights into the underlying genetic mechanisms responsible for specific traits in plants and animals, leading to a better understanding of their biology and potential for improvement.

The study of molecular genetics also enables the identification and characterization of genes associated with valuable traits in crops, such as drought tolerance, nutrient efficiency, and pest resistance.

By identifying and manipulating these genes, scientists can develop genetically modified organisms (GMOs) with enhanced traits, such as genetically modified crops with increased yield or improved nutritional content.

In conclusion, both genetics and molecular genetics have significant applications in agriculture. While traditional genetics focuses on selective breeding, molecular genetics allows for a more advanced and precise understanding and manipulation of genes. Together, these fields contribute to the development of improved crops and livestock for a more sustainable and efficient agricultural industry.

In the field of forensics, the study of genetics and molecular genetics has been instrumental in solving crimes and identifying individuals involved in criminal activities. Through the analysis of genetic material found at crime scenes, forensic scientists are able to compare and identify differences in the genetic profiles of suspects. This approach allows for a more reliable and accurate comparison of DNA samples, which is crucial in criminal investigations.

Molecular genetics, with its focus on the study of molecular structure and function, offers a more detailed and precise approach to forensic analysis. By examining specific genes and their variations, forensic scientists can establish a genetic profile of an individual, providing important clues in identifying suspects or victims.

Genetics, on the other hand, takes a broader perspective in the study of inherited traits and the genetic makeup of individuals. It involves the comparison of DNA sequences, studying inherited variations, and determining the likelihood of individuals carrying certain traits or diseases. In forensics, genetic analysis is crucial in establishing familial relationships, such as paternity or kinship, which can be helpful in suspect identification or victim identification.

By comparing the genetic profiles of individuals found at crime scenes with those in a database, forensic scientists can identify potential suspects or rule out individuals who may be wrongly implicated. This process involves a comprehensive analysis of DNA samples, including the identification of specific genetic markers that are unique to each individual.

While both genetics and molecular genetics play vital roles in forensic analysis, there are differences in their approaches and focus. Genetics offers a broader perspective on inherited traits, while molecular genetics provides a more detailed and in-depth analysis of genetic material. The comparison of genetic profiles and the identification of unique markers are common goals in both fields.

Overall, the study of genetics and molecular genetics is essential in forensic research and analysis. By using these approaches, forensic scientists are able to provide valuable evidence and insights in solving crimes, identifying suspects, and bringing justice to victims and their families.

In the study of genetics and molecular genetics, researchers employ different approaches and techniques to gain insights into the complex world of genes and their functions. The analysis of genetic information requires a combination of both genetic and molecular research methods.

Genetics research typically focuses on the inheritance and variation of genes among different individuals or populations. It often involves studying the physical traits, hereditary patterns, and genetic disorders in order to understand the role of genes in biological processes. Geneticists use various techniques such as pedigree analysis, linkage analysis, and genetic mapping to uncover the inheritance patterns of specific traits.

Molecular genetics, on the other hand, delves deeper into the molecular mechanisms behind genetic processes. This field employs advanced techniques to analyze DNA, RNA, and protein molecules. Researchers in molecular genetics use tools like polymerase chain reaction (PCR), DNA sequencing, and gene expression analysis to study the structure, function, and regulation of genes at the molecular level.

While genetics research focuses on broader patterns of inheritance and genetic variation, molecular genetics provides a more detailed understanding of the molecular events governing gene expression and regulation. Both approaches are crucial for unraveling the complexities of genetic information and its implications for health and disease.

In summary, genetics research and molecular genetics employ distinct research techniques to study genes and their functions. Genetics research takes a broader approach, focusing on inheritance patterns and genetic variation, while molecular genetics provides a deeper analysis of the molecular mechanisms behind genetic processes. Combining these two approaches enables researchers to gain a comprehensive understanding of genetics and its role in various biological processes.

The study of genetics and molecular genetics has had a significant impact on the field of evolutionary biology. By comparing the differences between genetic information and its molecular analysis, scientists have been able to gain a deeper understanding of the processes and mechanisms that drive evolution.

In traditional genetic study, scientists focus on the study of genes and their inheritance patterns within populations. This approach allows researchers to track the transmission of specific traits and determine how they are passed down through generations. However, this method does not provide detailed information about the molecular mechanisms that underlie genetic changes.

In contrast, molecular analysis takes a more detailed and precise approach. It involves the study of DNA and other molecules that make up the genetic material. Molecular techniques such as DNA sequencing, PCR, and gene expression analysis allow scientists to analyze the structure and function of genes at the molecular level. By understanding the molecular differences between individuals and species, researchers can gain insights into the evolutionary processes that shape biodiversity.

By combining the study of genetics with molecular analysis, scientists can compare genetic information across different species and populations. This comparative approach provides valuable insights into the evolutionary relationships between organisms and helps researchers reconstruct the evolutionary history of species.

Molecular genetics also allows researchers to study the impact of genetic variation and genetic changes on evolution. By analyzing DNA sequences, scientists can identify mutations and other genetic changes that occur over time. These studies help researchers understand the mechanisms of adaptation, speciation, and genetic drift, all of which play crucial roles in shaping evolution.

In summary, the integration of genetics and molecular analysis has revolutionized the field of evolutionary biology. This interdisciplinary approach provides researchers with a powerful tool to decipher the genetic and molecular basis of evolutionary processes. By understanding the impact of genetic variation and molecular differences, scientists can gain a deeper understanding of how species evolve and adapt in response to their changing environments.

In the field of genetics, the role of molecular genetics plays a crucial part in the process of genetic counseling. Genetic counseling aims to provide individuals and families with accurate information about the risk of genetic conditions and to support them in making informed decisions about their health and reproduction.

Molecular genetics involves the analysis of DNA, genes, and chromosomes to understand the underlying causes of genetic disorders. This study uses a different approach compared to traditional genetics, which focuses on studying the observable differences and traits in individuals.

One of the key roles of molecular genetics in genetic counseling is to analyze genetic variations that may contribute to an individuals risk of developing a genetic condition. Through advanced research techniques and technologies, molecular geneticists can identify specific changes in an individuals DNA sequence that could be associated with inherited diseases.

This analysis helps genetic counselors provide accurate information about the chances of the condition being passed on to future generations, providing individuals with a better understanding of their genetic risks. With this information, individuals can make informed decisions about family planning, reproductive options, and potential medical interventions.

Another important aspect of molecular genetics in genetic counseling is the comparison of DNA sequences. By comparing DNA sequences of individuals with and without certain genetic conditions, researchers can identify genetic variations that may contribute to the development of these disorders.

Comparative analysis helps in the early detection, diagnosis, and treatment of various genetic conditions, allowing individuals and families to access appropriate medical care and support. This approach enables genetic counselors to provide personalized genetic counseling, tailoring their recommendations and support based on the specific genetic profile of each individual.

Conclusion

The role of molecular genetics in genetic counseling is essential for understanding the underlying genetic factors contributing to inherited diseases. By analyzing genetic variations and comparing DNA sequences, molecular genetics provides valuable information that can guide individuals and families in making informed decisions about their health and reproduction. Through this approach, genetic counseling becomes more accurate, personalized, and effective in supporting individuals and families.

The comparison between genetics and molecular genetics in the context of disease diagnosis is crucial for understanding the approach and differences in the analysis of genetic disorders.

Genetics, as a field of study, focuses on the inheritance and variation of genes in organisms. It examines the role of genes in transmitting traits, including disease susceptibility. Genetic analysis involves studying the genetic makeup of individuals to identify mutations or alterations in specific genes that may contribute to the development of diseases.

In disease diagnosis, the study of genetics plays a significant role. Genetic tests can determine the presence or absence of specific genes associated with certain diseases. These tests help identify individuals who may be at risk or carriers for genetic disorders. Genetic counseling based on these findings can aid in making informed decisions about treatment options or preventive measures.

Traditional genetics primarily focuses on the study of inherited diseases caused by mutations in specific genes. It involves analyzing the presence of these mutations within families or populations to understand the pattern of disease transmission. This information is valuable in identifying affected individuals, predicting disease outcomes, and providing appropriate interventions.

Molecular genetics takes a more detailed and advanced approach to disease diagnosis compared to traditional genetics. It involves analyzing the structure and function of genes at a molecular level, including DNA sequencing and gene expression studies. The study of molecular genetics allows for a deeper understanding of the underlying mechanisms behind the development and progression of genetic disorders.

Molecular genetic analysis can identify specific genetic mutations or alterations that may contribute to disease susceptibility. It provides insights into the molecular pathways involved in disease development, allowing for targeted therapies and personalized treatment approaches. This approach also enables the identification of potential therapeutic targets and the development of novel treatment strategies.

In conclusion, both genetics and molecular genetics play essential roles in disease diagnosis. While genetics focuses on the inheritance and variation of genes, molecular genetics provides a more detailed analysis at the molecular level. Understanding the differences between these approaches is crucial for advancing our knowledge of genetic disorders and improving disease diagnosis and treatment.

In drug development, both genetics and molecular genetics play an important role in understanding the mechanisms of action of drugs and their potential effects on individuals. However, there are some differences in their approach and focus on research and study.

Genetics: a field of study that focuses on the inheritance and variation of genes in individuals and populations. It explores how genes influence the development, functioning, and characteristics of organisms. In drug development, genetics research aims to identify genetic variations that may affect the response to drugs, such as the presence of specific genes associated with drug metabolism or drug targets.

Molecular Genetics: a subfield of genetics that involves the study of DNA, RNA, and other molecules involved in genetic information and gene expression. It focuses on understanding the molecular mechanisms underlying various genetic processes, such as DNA replication, gene transcription, and protein synthesis. In drug development, molecular genetics research aims to unravel the molecular pathways and targets that drugs interact with in order to develop more targeted and effective treatments.

While genetics provides a broader perspective on the influence of genes on drug response and potential side effects, molecular genetics delves deeper into the specific molecular mechanisms through which drugs exert their effects. By combining the knowledge gained from genetics and molecular genetics, researchers can better understand the genetic basis of diseases and tailor drug therapies to individual patients.

By comparing the genetic profiles of patients who respond well to a drug with those who do not, researchers can identify genetic markers that predict drug response. This information can then be used to develop diagnostic tests that help identify individuals who are more likely to benefit from a particular drug or who may experience adverse reactions. Additionally, the study of molecular genetics can uncover new drug targets and pathways that can be exploited for drug development.

When comparing genetics and molecular genetics, it is important to consider the ethical implications of both approaches. While genetics is the study of genes and heredity, molecular genetics takes a more focused and analytical approach by examining the structure and function of genes at a molecular level. This difference in approach can lead to different ethical considerations in research and analysis.

In genetics, ethical considerations often revolve around issues such as privacy and informed consent. Researchers may need to handle sensitive information related to an individuals genetic makeup, which raises concerns about data privacy and confidentiality. Additionally, when conducting genetic studies, informed consent from participants is crucial to ensure that they fully understand the risks and benefits of participating in the research.

There is also the issue of genetic discrimination, where individuals may face discrimination based on their genetic information. This can have significant social and psychological impacts, highlighting the need for policies and laws to protect individuals from such discrimination.

In molecular genetics, ethical considerations are more focused on the research and analysis techniques used. Molecular genetics often involves the manipulation and modification of genetic material in a laboratory setting. This raises concerns about the potential risks and consequences of such manipulations. It is important to ensure that these techniques are conducted safely and in accordance with ethical guidelines to minimize any harm to both humans and other organisms.

Furthermore, the use of genetically modified organisms (GMOs) in molecular genetics research also raises ethical concerns. GMOs can have ecological implications and may raise questions about the potential long-term effects on the environment and other organisms.

In conclusion, while both genetics and molecular genetics share similarities in their study of genes, they have different approaches that can lead to distinct ethical considerations. Understanding and addressing these ethical concerns is essential to ensure the responsible and ethical advancement of genetic and molecular genetics research.

The analysis of genetics and molecular genetics is a field of research and study that poses several challenges for scientists. Both approaches involve the study of genes and the hereditary material, but they differ in their methodologies and scope. Understanding these challenges is crucial for advancing our knowledge in genetics and molecular genetics.

One of the current challenges in the field of molecular genetics is understanding the complexity of the molecular mechanisms that govern gene expression and regulation. Molecular genetics focuses on studying the individual molecules, such as DNA, RNA, and proteins, that make up the genetic material. This approach requires advanced techniques and technologies to analyze and manipulate these molecules, as well as computational methods to interpret the vast amount of data generated. The complexity of these molecular interactions presents a challenge for researchers in understanding the underlying mechanisms that control gene expression.

Another challenge in genetics and molecular genetics is the comparative analysis of data obtained from different organisms. While genetics traditionally involves studying specific traits and genes within a particular species, molecular genetics allows for a broader comparison across species. However, comparing genetic information between organisms can be difficult due to variations in gene structure, gene function, and regulatory mechanisms. Researchers need to develop standardized approaches and tools to compare and analyze genetic data from diverse organisms, which can help identify common patterns and evolutionary relationships.

In conclusion, the study of genetics and molecular genetics faces various challenges. The molecular complexity of gene regulation and expression requires advanced techniques and computational methods. Additionally, the comparative analysis of genetic data across different organisms calls for standardized approaches and tools. Overcoming these challenges is essential for advancing our understanding of genetics and molecular genetics and their applications in various fields, including medicine and agriculture.

The study of genetics and molecular genetics has provided valuable insights into the complexities and mechanisms of inheritance and genetic variation. However, there is still much to be discovered and understood in these fields. As technology continues to advance, new approaches and techniques are being developed that will further enhance our understanding of genetics.

One future direction is the use of comparative analysis to deepen our understanding of genetics. By comparing the genomes of different organisms, scientists can identify similarities and differences in their genetic makeup. This comparative approach allows for a more comprehensive understanding of how genetics shape biological traits and functions.

Furthermore, comparative analysis can help us uncover the evolutionary relationships between species. By examining the similarities and differences in their genetic information, scientists can reconstruct the evolutionary history of different organisms, shedding light on how life has evolved over millions of years.

Another future direction is the integration of molecular and genetic approaches. While genetics focuses on the study of inheritance and variation at the organismal level, molecular genetics delves into the underlying molecular mechanisms that drive these processes.

By combining these two approaches, researchers can gain a more comprehensive understanding of the genetic basis of traits and diseases. Molecular genetics provides the tools and techniques to examine the specific genes and molecules involved, while genetics provides the broader context and understanding of how these genes and molecules interact within an organism.

This integration of molecular and genetic approaches will allow for a more nuanced and sophisticated analysis of genetic variation and inheritance, providing valuable insights into the differences and similarities between individuals and populations.

In conclusion, the future of genetics and molecular genetics lies in the continued exploration of comparative analysis and the integration of molecular and genetic approaches. These advancements will further enhance our understanding of the complexities of genetics and pave the way for breakthroughs in fields such as personalized medicine and genetic engineering.

In the field of genetics, data analysis plays a crucial role in understanding the complexities of genetic information. Both genetics and molecular genetics employ different approaches in the study of genes and genetic variations.

In traditional genetics, the analysis focuses on studying traits and heredity patterns within populations or families. It involves observing and quantifying physical characteristics, as well as tracking the inheritance of specific traits through generations. This approach relies on family trees, Punnett squares, and statistical methods to analyze the data.

On the other hand, molecular genetics takes a more detailed and precise approach in analyzing genetic data. It involves studying the structure and function of DNA, genes, and proteins at the molecular level. This field has revolutionized the study of genetics by introducing techniques such as DNA sequencing and polymerase chain reaction (PCR).

Molecular genetics uses advanced laboratory techniques to isolate, amplify, and analyze specific regions of DNA. This allows researchers to identify and study genetic variations, mutations, and gene expression patterns. Data analysis in molecular genetics often involves complex algorithms, bioinformatics tools, and statistical methods.

By comparing genetics and molecular genetics, it becomes evident that the main difference lies in the level of detail and precision in data analysis. Traditional genetics provides a broader perspective on genetic traits and inheritance patterns, while molecular genetics offers a deeper understanding of the molecular mechanisms underlying genetic variations and gene functions.

Overall, the comparison between genetics and molecular genetics highlights the evolving nature of genetic studies. While both approaches contribute to our understanding of genes and heredity, molecular genetics allows for a more in-depth analysis of genetic data, paving the way for new discoveries and advancements in the field.

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Comparing Genetics and Molecular Genetics: What's the Difference?

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University of Colorado Anschutz Medical Campus-Led Team Receives Up to $46 Million to Develop Innovative Treatment to Cure Blindness – University of…

Friday, December 6th, 2024

University of Colorado Anschutz Medical Campus-Led Team Receives Up to $46 Million to Develop Innovative Treatment to Cure Blindness  University of Colorado Anschutz Medical Campus

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University of Colorado Anschutz Medical Campus-Led Team Receives Up to $46 Million to Develop Innovative Treatment to Cure Blindness - University of...

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Meet our Team | Animal Medical Center of Loudoun

Monday, October 14th, 2024

Rocky Mountains or Smoky Mountains?All Mountains!

Garden or Wild?Garden and Wild!

Know Where Youre Going or Just Walk Around?Just Walk Around

Swim in a River or Swim in a Pool?Swim in a River

Butterfly or Lightning Bug?Dont Make Me Choose Between a Butterfly and Lightning Bug Ok, Butterfly

Geometric or Organic?Organic

Eagle or Owl?Owl

Full Moon or Crescent Moon?Full Moon

Walk the Dogs or Feed the Chickens?Walk the Dogs(But the Chickens Must Be Fed)

Dinner Party or Watch TV?Dinner Party

Leather or Cashmere?Cashmere

Hitting the Slopes or Cozy Fire?Cozy Fire

Flea or Farmers Market?Farmers Market

By the Mountains or Sea?Mountains with Sea Vacations

Imagine a veterinarian that is a skilled medical practitioner that utilizes cutting-edge diagnostics and medical treatments, but also offers a more subtle, holistic treatment choice someone who understands that your lifestyle choices carry over into the way you care for your pet. The ability to mesh Western and Eastern medical approaches is highly unusual, but certainly welcome in todays veterinary community. If you are looking for a veterinarian that offers this unique ability, then we have the doctor for you. Dr. Pamela Grasso balances the best of both medical worlds.

Having earned a reputation as a kind-hearted spirit with a natural sense of compassion and a special touch for healing, Dr. Grassos clients offer a continuous stream of new patient referrals. It is interesting to see a trained Western medical practitioner embrace holistic medicine so easily. When asked how she progressed from modern medicine to the ancient Chinese practices, Dr Grasso explains, I referred some of my arthritic patients to a veterinary acupuncturist because they either couldnt tolerate pain medications or were not responding to treatment. The pet owners began raving about acupuncture and how great their dogs felt after just a few visits.

Dr. Grasso attended Bucknell University and earned a Bachelors Degree in Biology (and a minor in Chemistry) in 1983. She continued her education at Pennsylvania State University where she earned a Masters Degree in Veterinary Science in 1987. She became a veterinarian in 1991, after obtaining her Doctorate of Veterinary Medicine from the North Carolina State University. After practicing Western veterinary medicine for several years, she decided to learn a new approach. She received her Certification of Veterinary Acupuncture in 2000. In 2002, she attended a comprehensive course in Western Herbal Medicine. In 2004, she completed all modules the IVAS Traditional Chinese Herbal Medicine training. In 2007, she completed the Professional Course in Veterinary Homeopathy with Richard H. Pitcairn, DVM. In 2008, she completed Advanced Veterinary Homeopathy training.

Dr. Grasso is an active member of the American Veterinary Medical Association (AVMA), International Veterinary Acupuncture Society (IVAS), Veterinary Botanical Medical Association (VBMA), American Holistic Veterinary Medical Association (AHVMA), American Botanical Council (ABC), and American Academy of Veterinary Acupuncture (AAVA), The Academy of Veterinary Homeopathy (AVH), and is USDA Accredited.

Dr. Grasso shares her home with her Irish Wolfhounds, cats, chickens, and horse. She is actively involved in Irish Wolfhound rescue. In her spare time, she enjoys the craft of stained and hot glass. When the weather permits, she loves to be outdoors hiking, gardening, camping, or kayaking.

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Meet our Team | Animal Medical Center of Loudoun

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Meet our Veterinarians | Animal Medical Center of Loudoun

Monday, October 14th, 2024

Rocky Mountains or Smoky Mountains?All Mountains!

Garden or Wild?Garden and Wild!

Know Where Youre Going or Just Walk Around?Just Walk Around

Swim in a River or Swim in a Pool?Swim in a River

Butterfly or Lightning Bug?Dont Make Me Choose Between a Butterfly and Lightning Bug Ok, Butterfly

Geometric or Organic?Organic

Eagle or Owl?Owl

Full Moon or Crescent Moon?Full Moon

Walk the Dogs or Feed the Chickens?Walk the Dogs(But the Chickens Must Be Fed)

Dinner Party or Watch TV?Dinner Party

Leather or Cashmere?Cashmere

Hitting the Slopes or Cozy Fire?Cozy Fire

Flea or Farmers Market?Farmers Market

By the Mountains or Sea?Mountains with Sea Vacations

Imagine a veterinarian that is a skilled medical practitioner that utilizes cutting-edge diagnostics and medical treatments, but also offers a more subtle, holistic treatment choice someone who understands that your lifestyle choices carry over into the way you care for your pet. The ability to mesh Western and Eastern medical approaches is highly unusual, but certainly welcome in todays veterinary community. If you are looking for a veterinarian that offers this unique ability, then we have the doctor for you. Dr. Pamela Grasso balances the best of both medical worlds.

Having earned a reputation as a kind-hearted spirit with a natural sense of compassion and a special touch for healing, Dr. Grassos clients offer a continuous stream of new patient referrals. It is interesting to see a trained Western medical practitioner embrace holistic medicine so easily. When asked how she progressed from modern medicine to the ancient Chinese practices, Dr Grasso explains, I referred some of my arthritic patients to a veterinary acupuncturist because they either couldnt tolerate pain medications or were not responding to treatment. The pet owners began raving about acupuncture and how great their dogs felt after just a few visits.

Dr. Grasso attended Bucknell University and earned a Bachelors Degree in Biology (and a minor in Chemistry) in 1983. She continued her education at Pennsylvania State University where she earned a Masters Degree in Veterinary Science in 1987. She became a veterinarian in 1991, after obtaining her Doctorate of Veterinary Medicine from the North Carolina State University. After practicing Western veterinary medicine for several years, she decided to learn a new approach. She received her Certification of Veterinary Acupuncture in 2000. In 2002, she attended a comprehensive course in Western Herbal Medicine. In 2004, she completed all modules the IVAS Traditional Chinese Herbal Medicine training. In 2007, she completed the Professional Course in Veterinary Homeopathy with Richard H. Pitcairn, DVM. In 2008, she completed Advanced Veterinary Homeopathy training.

Dr. Grasso is an active member of the American Veterinary Medical Association (AVMA), International Veterinary Acupuncture Society (IVAS), Veterinary Botanical Medical Association (VBMA), American Holistic Veterinary Medical Association (AHVMA), American Botanical Council (ABC), and American Academy of Veterinary Acupuncture (AAVA), The Academy of Veterinary Homeopathy (AVH), and is USDA Accredited.

Dr. Grasso shares her home with her Irish Wolfhounds, cats, chickens, and horse. She is actively involved in Irish Wolfhound rescue. In her spare time, she enjoys the craft of stained and hot glass. When the weather permits, she loves to be outdoors hiking, gardening, camping, or kayaking.

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Center for Nanomedicine – Johns Hopkins Medicine

Sunday, October 6th, 2024

The Johns Hopkins Center for Nanomedicine (CNM) brings together engineers, scientists, and clinicians working together under one roof on translation of novel drug and gene delivery technologies. The focus is to overcome major challenges to drug efficacy, including biological barriers to delivery, patient compliance, and toxicity. Furthermore, we educate and train the next generation of researchers to innovate at the interface of engineering, medicine, and the life sciences in the development of drug delivery technologies with potential for clinical implementation. We believe that collaboration and commitment to diversity are key in having the maximum impact on human health.

Nanomedicine is defined as the medical application of nanotechnology. In the CNM, we focus on harnessing nanotechnology to more effectively diagnose, treat, and prevent various diseases. Our entire bodies are exposed to the medicines that we take, which can lead to unpleasant side effects and minimize the amount of medicine that reaches the places where it is needed. Medications can be more efficiently delivered to the site of action using nanotechnology, resulting in improved outcomes with less medication.

We design our nanotechnology-based platforms for clinical translation. What this means is that we strive for innovative simplicity and the use of components that have a history of medical safety, so that our nanomedicines can be tested in clinical trials and developed into useful products. To this end, we often design our platforms to mimic nature or select our systems based on how they naturally distribute in the body. To date, our faculty have founded more than 10 start-up companies, resulting in several FDA-approved products and others being tested in clinical trials.

Focus areas: Glaucoma, age-related macular degeneration, diabetic retinopathy, retinitis pigmentosa, diabetic macular edema, uveitis, dry eye disease, corneal neovascularization, corneal graft rejection, thyroid eye disease, cataract surgery, glaucoma surgery, ocular trauma

Focus areas:Immunotherapy, cancers of the pancreas, cervix, ovary, lung, brain, bladder, and colorectum

Focus areas: COVID-19, inflammatory bowel disease, pancreatitis, liver cirrhosis, various cancers, intrauterine inflammation, atherosclerosis, arthritis

Focus areas: Cystic fibrosis, asthma, chronic obstructive pulmonary disease, lung cancer

Focus areas: Preterm birth, intrauterine inflammation, infections,in vitrofertilization, reproductive tract cancers, contraception

Focus areas:Brain tumors, cerebral palsy, neonatal stroke, traumatic brain injury, autism spectrum disorders, Parkinsons disease, Alzheimers disease

The CNM team includes basic science and clinical faculty, research staff, postdoctoral fellows, graduate students, and undergraduates. Our goal is to train, mentor, and promote inclusive and supportive research environments.

In addition to a rigorous and broad training in unbiased experimental design, methods, data analysis, interpretation, and reporting, we strive to support our trainees in career and professional development. We proudly support diverse career goals, and our alumni have gone on to careers in academia, industry, regulatory, venture capital, consulting, science communication, policy, law, and medicine. We participate in numerous training programs that promote and develop trainees from low income and underrepresented groups in biomedical research.

Kannan Lab

Research Specialist, Ensign Lab

Research Specialist, Ensign Lab

Research Specialist, Ensign Lab

Research Specialist

Lab Manager

Research Technologist

Lab Manager

Research Engineer

Hanes Lab

Ensign Lab

Mahdi Forouharshad, Ph.D.

Thanuia Marasarakottige Yogananda, Ph.D.

Balachandra Chenikkayala, Ph.D.

Durgadas Cherukaraveedu, Ph.D.

Ensign Lab

Hanes Lab

Ensign Lab

Ensign Lab

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Hanes Lab

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Center for Nanomedicine - Johns Hopkins Medicine

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Emerging Applications of Nanotechnology in Healthcare and Medicine

Sunday, October 6th, 2024

Abstract

Knowing the beneficial aspects of nanomedicine, scientists are trying to harness the applications of nanotechnology in diagnosis, treatment, and prevention of diseases. There are also potential uses in designing medical tools and processes for the new generation of medical scientists. The main objective for conducting this research review is to gather the widespread aspects of nanomedicine under one heading and to highlight standard research practices in the medical field. Comprehensive research has been conducted to incorporate the latest data related to nanotechnology in medicine and therapeutics derived from acknowledged scientific platforms. Nanotechnology is used to conduct sensitive medical procedures. Nanotechnology is showing successful and beneficial uses in the fields of diagnostics, disease treatment, regenerative medicine, gene therapy, dentistry, oncology, aesthetics industry, drug delivery, and therapeutics. A thorough association of and cooperation between physicians, clinicians, researchers, and technologies will bring forward a future where there is a more calculated, outlined, and technically programed field of nanomedicine. Advances are being made to overcome challenges associated with the application of nanotechnology in the medical field due to the pathophysiological basis of diseases. This review highlights the multipronged aspects of nanomedicine and how nanotechnology is proving beneficial for the health industry. There is a need to minimize the health, environmental, and ethical concerns linked to nanotechnology.

Keywords: nanotechnology, nanobiotechnology, nanomedicine, medical applications, diagnosis, disease treatment, drug-delivery, healthcare

The world is theorized to have accidentally formed via the Big Bang that occurred from an unstable microscopic-sized energized particle (atom). A single bit created an entire universe, and now scientists are working again on similar small particles to create marvels of science. From here, the world of nanoscience has arrived and taken a firm place in every aspect of science and technology [1]. The vision for nanotechnology was presented by Nobel Prize-winning physicist Richard P. Feynman, who proposed the application of more significant objects and mechanistic tools at a smaller tool and particle scale, as he believed that there is plenty of room at the bottom [1,2]. Nowadays, apart from physicists, scientists from multiple fields believe that in the future, nanoscale manufacturing technologies and instrumentation such as nanomachines, robotics, nanomedicine, and diagnostic devices, among many others, will bring grand biomedical miracles to the world of medicine and other industries [3,4,5,6,7].

Nanoscale pertains to the size of one-billionth or 109 m of a material. A new scientific field of science in the form of nanotechnology was created because it was observed that materials, products, and devices developed from nanoscale particles almost always exhibit properties different from those of large-scale bulk materials. This follows the basic principles of physics and chemistry that as the state of matter is composed of atoms, any changes in atomic size, shape, and arrangement directly affect the materials properties [7,8]. Scientists think that nanotechnology is the future of science and thus they are looking forward to benefitting from the application of nanotechnology in almost every possible way. The unique properties and behavioral features of nanoscale products have also drawn the attention of clinicians, physicians, and biological researchers [9,10]. The effort is on its way to applying unique quantum phenomena at the nanoscale to the fields of medicine, biomedical sciences, bioengineering, food technology, biochemistry, biophysics, and other disciplines of biology and medicine [10,11,12,13].

Forty years of revolutionary interaction among biology, medicine, and nanotechnology have led to present-day nano-biotechnology, which is now showing progressive application in multiple aspects of the medical field [14]. From disease detection to treatment, many medical issues such as disease diagnosis, drug discovery, personalized medical procedures, cancer treatment, pharmaceutical discoveries, as well as the latest medical tools and procedures, are now improving on the uses of nano-biotechnology [15]. Similar to regular vaccination approval, nano-based medicine and nanovaccines are also obtaining regular medical approval with the passage of time. Various nanotechnology-based diagnostic kits such as nanosensors, nanoparticle-based imaging agents, nanoparticle-based PCR Assays, Lab-on-a-Chip devices, along with modern drugs and medicines such as nanoparticle-based drug delivery vehicles, liposomal formulations and polymeric nanoparticles, Nanomedicines (such as Abraxane (nanoparticle albumin-bound paclitaxel) and Doxil (pegylated liposomal doxorubicin)), nanotechnology in gene therapy, nanoparticle-based vaccines, and antimicrobial agents, etc. are being commercialized for research and clinical usage [16].

Nanomedicine is a broad-spectrum field of science and technology that unites multiple streams of medical applications such as disease treatment and diagnosis, disease prevention, pain relieving technologies, human health improvement medicine, nanoscale technology against traumatic injury, and treatment options for diseases [12,15]. Thus, an interdisciplinary approach is being adopted to apply the outcomes of biotechnology, nanomaterials, biomedical robotics, and genetic engineering combined under the broad category of nanomedicine [17]. On a broader level, nanoscaling of medical technologies provides efficiency, a rapid response rate, and functional effectiveness in most biological and chemical processes used to manufacture medical materials. Thus, research provides constant hope for the upcoming new applications of nanomedicine [12,18].

In this review article, comprehensive analyses have been carried out to examine the application of nanotechnology specifically in the field of medicine. The most advanced form of nanotechnological applications have been highlighted with a slight emphasis on the previous uses of nanotechnology in the past few years of the 21st-century. Some modern medical applications, such as diagnostics, nanomedicine, regenerative medicine, and personalized targeted therapies, have also been included to bring into account the latest nanomedical applications.

Diagnostic sciences are now using nanodevices for early and rapid disease identification for further medical procedural recommendations. It also utilizes nanotechnology for the predisposition of disease at the cellular and molecular level to develop insights into treatment options [16]. Nanotechnology has the potential to revolutionize the field of healthcare diagnostics by improving the accuracy, sensitivity, and speed of medical tests [18]. One of the profound applications includes nanoparticle-based diagnostic imaging, in which nanoparticles can be attached to specific biomarkers to enhance imaging modalities such as magnetic resonance imaging (MRI), computerized tomography (CT) scans, and positron emission tomography (PET) scans, making them more sensitive, accurate, and specific [19]. Similarly, nanotechnology-enabled point-of-care diagnostic tests can quickly and accurately detect infectious diseases, cancers, and other illnesses, enabling timely treatment and prevention [9,19].

Biosensors are yet another dimension of application in which nanotechnology has enabled the development of highly sensitive biosensors that can detect even low levels of biomolecules in bodily fluids such as blood and urine, facilitating early detection and disease management [20,21]. Similar applications come in the form of microfluidic devices that incorporate nanomaterials and can be used to isolate and analyze specific cells, proteins, and genetic material, providing rapid and accurate diagnosis of diseases [19,22]. Another use may involve nanopore sequencing, which is a novel technology that uses nanopores to detect the sequence of DNA or RNA molecules, allowing for rapid and accurate diagnosis of genetic disorders such as cancer and genetic diseases [23].

Recent advances show that nanomedicine can be used in in vitro diagnostics sciences to increase the efficiency and reliability of disease apprehension [24]. This is achieved via nanodevices at the subcellular level, with samples prepared from human tissue, cell culture, body fluids, etc. [19,25,26]. In in vivo diagnostics, the nanomedicine approach is being used to develop devices capable of working, responding, and modifying within the human body with the sole purpose of early diagnosis of any irregularities in the human body that could lead to toxicity or tumor development events [22,27]. A few types of nanoparticles that are currently in use for diagnostic purposes include paramagnetic nanoparticles, nanocrystals, quantum dots, nanoshells, and nanosomes [28,29]. Overall, nanotechnology has enormous potential in healthcare diagnostics and is expected to play a significant role in the development of personalized medicine.

Nanotechnology and Lab-on-Chip Technology have revolutionized the field of healthcare by offering innovative solutions for disease diagnosis, personalized treatment, and drug delivery [15]. The combination of these two technologies has led to the development of advanced diagnostic tools that are faster, more accurate, and more cost-effective than traditional diagnostic methods [30]. Lab-on-Chip technology is making progress in different fields of science; for example, it is being considered for use against viral and cancerous diseases [15,24]. The whole process revolves around analyzing genetic information at the cellular level [30]. Advanced procedures of gene sequencing and body fluid sampling have further assisted in revolutionizing nanotechnology in service of cures for diseases that were previously unimaginable [31,32].

Together, these two technologies have led to the development of Lab-on-Nanoparticles, which are small devices that can perform multiple functions, including diagnostics, drug delivery, and monitoring of various health conditions [31,32]. These devices are made up of nanoscale materials that can detect and respond to changes in the body, allowing for real-time monitoring and personalized treatment [26]. One of the significant applications of nanotechnology and Lab-on-Chip Technology in healthcare is cancer diagnosis [20,21]. Nanoparticles can be designed to target cancer cells, allowing for early detection and treatment [33]. Lab-on-Chip devices can also be used to diagnose various health conditions, including infectious diseases, genetic disorders, and metabolic disorders [32,34].

The use of nanotechnology and Lab-on-Chip Technology in healthcare has also led to the development of advanced drug delivery systems [31]. Nanotech systems such as nano-Liposomes can target specific cells or tissues in the body, enhancing drug efficacy and reducing side effects [28,35]. Moreover, viral detection is considered a feature that will be linked to future generations of nanoscale diagnostic devices. Such devices are expected to enable the detection of the release of medications in the organs of the body, which will help in the calculation of treatment efficiency and efficiency rates [36]. In simple terms, nanotechnology is trying to increase the pharmacokinetic and pharmacodynamic properties of drugs to stay longer inside the body, work faster and more efficiently, and at essential sites [37].

A brief overview of nanotechnological applications in pharmaceutical sciences has been covered in the following section with a diagrammatic representation in .

Applications of nanotechnology in pharmaceutical sciences.

Nanoscience has revolutionized the pharmaceutical industry by enabling the production of improved therapeutic drugs with enhanced efficacy and lower toxicity. Nanoparticles can improve the pharmacokinetics of drugs by increasing their solubility, stability, and bioavailability [38]. They can also target specific tissues and cells, reducing side effects and enhancing their efficacy [25]. The nanoscale size and unique physicochemical properties of nanoparticles demand precise specifications in terms of drug dose and administration [39,40]. The dose of nanoparticles depends on various factors such as their size, shape, surface properties, and the method of administration [40]. For instance, oral administration may require a higher dose to achieve the same effect as intravenous administration due to the differences in absorption and biodistribution [40,41].

Furthermore, nanoparticles have complex pharmacokinetics and dynamic behavior in vivo, requiring a careful consideration of their dose regimen [40]. Researchers need to determine the optimal dose range, frequency, and duration of nanoparticles to achieve their therapeutic goals while minimizing adverse effects [41,42]. In the past, medical studies have resulted in very advanced treatment options; however, there is still a gap in effectively neutralizing drug overdoses. The use of nanoparticles as absorbents of toxic drugs is a feature being taken into account to create a rich method of drug absorption in the medical sciences [40,41,42]. The design of nanosponge-type substances is on the way to absorb unnecessary toxic dosages of drugs in blood to reduce the side effects of drug overdoses and treat ailments from body fluids [43]. Such antiviral drug absorbents have been introduced by researchers that work as nanoscale molecules to render anticancer and antiviral nucleoside analogs by linkage with squalene [44]. These nano-assemblies work as superior anti-cancerous molecules to treat human cancer cells that have yet to be developed beyond in vitro studies [45]. In summary, the development of nanomedical products requires careful consideration of the dose and administration of nanoparticles to ensure their efficacy and safety. The nanoscience community must collaborate with regulatory agencies to develop guidelines for nanomedicine testing to ensure their safety and efficacy.

Nanotechnology has revolutionized the field of drug delivery by providing an effective and targeted delivery of drugs, minimizing side effects, and increasing the therapeutic efficacy of drugs. The application of nanotechnology in drug delivery involves the use of nanoparticles that are designed to carry drugs and deliver them to the desired site of action [46]. The use of nanotechnology in drug delivery has several advantages. First, it allows for targeted and controlled delivery of drugs to specific sites in the body, such as tumors, inflamed tissue, and infected areas [46]. This reduces the amount of drugs required and minimizes side effects. Secondly, nanoparticles can improve the solubility and stability of drugs, making them more effective in treating diseases [47]. Thirdly, nanotechnology can increase the bioavailability of drugs by enhancing their absorption and distribution in the body. This allows for lower doses of drugs to be used, resulting in reduced toxicity [48,49].

Drug delivery technologies are also being given full consideration to be modified as per the new rules of nanoscaling. Some kinds of medical nanorobots are in line to be used for medicine delivery [32]. These materials swim across veins and carry drugs to specific sites. These aspects are being used for antitumoral responses of drugs [48]. Scientists are even working on performing wireless intracellular and intranuclear nanoscale surgeries against multiple malignancies and diseases [46,48]. Marvelous scientific arrangements are being carried out in the form of manufacturing and testing mechanical red blood cell technologies called respirocytes. Nanorobotics share the potential to deliver 200+ times more oxygen to body tissues as compared to natural red blood cells [49,50]. This could make one think about the potential of nanotechnology to be utilized for the diagnosis and treatment of various blood-linked disorders and their cure in the future [50]. In conclusion, the application of nanotechnology in drug delivery has revolutionized the field of medicine. It has provided an effective and targeted delivery of drugs, minimized side effects, and increased the therapeutic efficacy of drugs. The future of drug delivery lies in the continued development of nanotechnology-based drug delivery systems.

DNA-based drug delivery devices have been introduced in the past few years, such as DNA guns and DNA vaccines. Based on similar principles, an emerging field of DNA nanotechnology is being introduced in the nanomedicine industry [51]. These medical tools allow for the self-assembly of nanostructures and molecules that ultimately enhance drug targeting and reduce the toxicity associated with these drugs. With such technology, toxicity measures can be easily dealt with in diseases such as cancer, where the major issue is the drug toxicity associated with chemotherapeutic drugs [24,51].

The latest advances in research indicate that modern programing optimization and in silico approaches are being adopted to design DNA nanostructures with precise size, structure, surface chemistry, and functioning properties against specific diseases [22,52]. The effort is also to create personalized targeted drug therapies using nanotechnology-based DNA medicine [51]. Efficient drug biomolecules, such as doxorubicin and CpG oligonucleotides, have been successfully amalgamated with DNA-based nanostructures to increase cellular intake efficiency [53]. The future holds the potential to create RNA-based medication using principles similar to those employed in DNA-based medication [54].

Nanobiotechnology and gene therapy are two fields that often intersect in the development of innovative therapeutic approaches for the treatment of various diseases. In gene therapy, DNA molecules are introduced into the patients cells to replace defective or missing genes, with the aim of treating genetic disorders and other diseases [55]. One application of nanobiotechnology in gene therapy is the use of nanoparticle-based delivery systems to transport therapeutic genes to target cells [41,56]. These nanocarriers protect the DNA molecules from degradation and enhance their ability to penetrate the cell membrane, increasing the efficacy and safety of gene therapy [53,56].

Other nanobiotechnology approaches that support gene therapy include the development of gene editing technologies that use nanoscale tools to precisely modify DNA sequences and correct genetic mutations [57]. Additionally, nanoparticle-based sensors can be used to monitor gene expression and other molecular events in real-time, providing valuable information for personalized medicine [32]. Modern therapeutic concepts including gene therapy and molecular DNA-based therapies are already being practiced in healthcare and the arrival of nanotechnology has forwarded further advances in it [58]. Since the very basis of working gene therapy is at the molecular level of disease prevention and genetic adjustments, nanoscale technology plays a vital role in gene therapy [58].

Gene therapy processes are being modified to attach different kinds of biodegradable and non-biodegradable organic and inorganic particles fabricated with nano-assemblies. These structural combinations help bind DNA and access it across cellular surfaces [59]. Moreover, polymer-based nanoparticle mixtures are also prepared for intravenous drug injections. These modified technologies are a gateway to further advances in nanogenetic therapies [60]. Overall, the integration of nanobiotechnology and gene therapy is expected to lead to advanced treatments for a wide range of diseases, including cancer, genetic disorders, and infectious diseases.

Polyplex micelles are a type of nano-sized structure that are formed by the self-assembly of cationic polymers with nucleic acids, such as small interfering RNA (siRNA) or plasmid DNA (pDNA) [61]. These polyplex micelles have attracted significant attention for their potential in gene therapy and as drug delivery systems. In the context of tumor treatment, various polyplex micelle-based strategies using siRNA and pDNA have been studied. siRNA is an RNA molecule that is used to specifically target and knock down the expression of disease-related genes [62,63]. Plasmid DNA (pDNA) is a circular DNA molecule that can carry therapeutic genes to the target site. Polyplex micelles can encapsulate siRNA or pDNA within their core, protecting them from degradation and facilitating their delivery to tumor cells [63,64]. Additionally, the cationic nature of the polyplex micelles allows for electrostatic interactions with the negatively charged cell membrane, promoting their uptake by tumor cells [64].

These polyplex micelle-based strategies have been investigated for the treatment of various tumors, including pancreatic adenocarcinoma [63]. Pancreatic adenocarcinoma is a particularly challenging type of solid tumor resistant to many conventional treatment options. By using polyplex micelles, siRNA or pDNA can be delivered specifically to the tumor cells, enabling targeted gene therapy or enhancing the efficacy of chemotherapeutic drugs [64]. Thus, nanotechnology, specifically polyplex micelles, offers a promising approach for delivery of siRNA or pDNA to tumors such as pancreatic adenocarcinoma. These micelles can protect genetic material, promote cellular uptake, and potentially enhance the effectiveness of treatments for intractable solid tumors [65].

Nanomedicines are largely produced through chemical and physical methods of downgrading particles up to micro- and nanoscales. However, with the concerns of environmental and toxic health impacts, nanomedicine is now employing the concept of green chemistry and green engineering into the manufacturing of nanobiomedicine [66]. The purpose of this green technology is to create eco-friendly nanoassemblies with less environmental and health-related negative impacts [66]. Subsequently, the combination of green nanoassemblies with drugs, vaccines, or diagnostic markers will be the next step to propel the field of green nanomedicine. Many inorganic nanoassemblies have been introduced to the market and manufactured on the principles of green engineering and nanotechnology [67]. Some examples may include gold and silver nanoparticles, quantum dots, organic polymeric nanoparticles, mesoporous silica nanoparticles, dendrimers, nanostructured lipid carriers, solid lipid nanoparticles, etc. [66,67,68].

These nanoassemblies are attached with drugs, DNA molecules, or specific enzymes, proteins or peptides for further handling in nanomedicine purposes [66]. However, the need is to establish research studies that demonstrate the difference and effectiveness level of nanomedicine produced using normal bioengineering against that of manufacturing of nanomedicines through the elaborative principles of green bioengineering [66,67,68]. This will allow scientists to opt for the best manufacturing conditions for nanoassemblies in the future.

The causative agents of viral, bacterial, and other microscopic diseases work at the microscopic level; therefore, the best way to fight against them is at the nanoscale. Nanotechnology is thus the gateway to the cure and diagnosis of a wide range of viral, bacterial, and fungal diseases [69]. Although traditional Greek medicinal practices have been using metals such as silver to cure diseases for a long time, an updated version of nanoscale-based material conversion has been shown to improve the efficiency of such traditional and modern medication options [70]. One such study carried out by Nycryst Pharmaceuticals (Canada) showed that nanosized silver particles are more reactive to cure burn or wound as they easily penetrate the skin at some small scale [71].

The genomic and proteomic fields are already contributing much to the elucidation of molecular insights into disease, and with the assistance of nanotechnology, new opportunities are being put in the hands of researchers to create powerful diagnostics tools with the power of genetic elucidation of irregularities at the level of the gene [72]. Research indicates that soon, nanotechnology-based diagnostic and treatment options will be available for preventive and regenerative medicine with targeted and personalized therapy potential against pathogenic and pathophysiological diseases [70,71,72,73]. All these benefits are coupled with the cost-effective and time-saving aspect of this new technology.

There are several barriers or issues associated with nanoparticles in terms of delivery efficiency and clinical translation. The accumulation of nanocarriers in organs of the reticuloendothelial system, especially the liver, poses a significant challenge for clinical translation as it captures a majority of the injected dose, hindering the delivery of an adequate dose to the targeted disease site and potentially causing toxicity concerns [74]. Researchers have developed various approaches to address this issue, including preconditioning macrophages with chloroquine, saturating the reticuloendothelial system organs with drug-free nanocarriers, and transient stealth-coating scavenger cells to enhance the efficiency of drug-loaded nanoparticles reaching the diseased tissue [75,76,77]. Additionally, the incorporation of targeted cellular on the surface of nanocarriers such as those applying the do not eat us strategy, helps evade capture by the reticuloendothelial system, improving the accumulation of nanodrugs at the desired site [73,76].

On the other hand, surface shielding of nonionic hydrophilic polymers such as PEG on nanocarriers reduces cellular uptake and endosomal escape, resulting in poor delivery efficiency despite improving colloidal stability and stealth in a biological environment [77]. To overcome this stealth dilemma, targeting ligands are strategically placed at the distal end of the PEG segments to facilitate specific ligand receptor-mediated uptake [78]. Another strategy involves wrapping anionically charged polymers on positively charged mRNA-polyplexes to promote endosomal escape by converting them into positively charged polymers in response to the acidic pH of the endo/lysosomal compartments [79].

The use of messenger RNA (mRNA)-loaded lipid nanoparticles is limited by their hepatic protein expression, even when administered locally through intramuscular and intratumor injections [80]. Minimizing the off-target hepatic expression would be advantageous for protein replacement therapies and cancer immunotherapies. One approach involves incorporating microRNA target sites in therapeutic mRNAs to selectively prevent their expression in the liver [80]. Some other generalized barriers associated with nano-based drug delivery mechanisms are included in . It is important to note that although nanoparticles face these barriers and issues, significant advancements are being made in addressing them, bringing us closer to their successful clinical translation.

Barriers associated with nano-based drug delivery.

Nanotechnology is the science of creating and manipulating materials at the molecular and atomic levels. Bone regeneration technology creates new bone tissue, or helps existing bone tissue heal, with the use of materials that promote bone growth [81]. Nanotechnology is increasingly used in bone regeneration technology to create better, more precise and targeted materials for promoting bone growth [80]. For example, researchers are exploring the use of nanoparticles to deliver drugs or other molecules that promote bone growth directly to the areas that need them, improving the effectiveness of the treatment [80].

Nanoparticles can also be used to create scaffolds that mimic the structure of bone, which can help guide new bone growth and aid in bone regeneration. Additionally, advances in 3D printing technology that uses nanoscale materials can be used to create highly precise and customized implants for bone regeneration [81]. Bone weakening and dysfunction is a widespread problem and this has been marked by nanotechnologists as an issue of the utmost importance when linking nanotech to medicine. Some studies are being carried out regarding bone formation and structuring with the help of nanotechnology [80,81]. Scientists are trying to develop bone graft substitutes in the form of nanostructured materials with similar properties to be accepted by body and organ tissues. If these studies succeed, they will bring a new wave of regenerative technology to cure damaged bones and broken muscular fragments [82].

Principle investigation on biomineralization is being carried out to reduce the particle size of bone materials that could be coupled with its crystalline properties to be embedded into collagen fibers [80]. The purpose is to create a penetrating composition in damaged bone areas with specific mechanical properties to revolutionize the field of osteology and bone tissue engineering [80,81]. Similar studies are being carried out to make artificial joints, nanoscale collagen-mimicking coatings for knees and hips that act to stabilize the process of bone formation by osteoblasts [83,84]. Overall, the use of nanotechnology in bone regeneration technology holds great promise for improving the outcomes of bone repair and regeneration, including faster healing times, improved bone strength, and reduced complications.

Regenerative medicine is an interdisciplinary field of medical applications in which the benefits of cell therapy and tissue engineering methods are well fabricated to device mechanisms for the treatment, maintenance, improvement, and reparation of damaged and dead cells, tissues, and organs [73]. Previously, it was difficult to deal with the body at the cellular level but with the emergence of nanoscale technology, a huge opportunity has become available in the form of regenerative medicine to interact with cells and their components so that the linked cellular responses and extracellular material production can be controlled [80]. Tissue repair has been greatly upgraded with the powerful tissue regeneration abilities of nanoassemblies. These technologies are being directed for cellular adhesion, migration, differentiation, and other mechanical aspects that initiate tissue regeneration [85].

Exploration in the field of nanomedicine is going on to manufacture nanoscale materials, such as gold and silver nanoparticles, dendrimers, nanorods, carbon buckyballs, nanoshells, nanocubes, and many other forms of nanoparticles [73,79]. Each is specific to its linked properties, which can be directly utilized in targeted tissues and organs. Multiple research groups are working worldwide to explore the diagnostic, therapeutic, anti-viral, antifungal, and most importantly anticancerous properties of these nano-agents [70,72,86]. Progress shows that soon, a world of nanotechnology will bring a revolution to the treatment options for incurable diseases such as cancers, for which early diagnosis through nanotechnology is already on board and has been successfully explored [73,86].

A brief overview of nanotechnological applications in surgery is covered in the following section with a diagrammatic representation in .

Applications of nanotechnology in surgery.

Surgical nanorobotics involves the development and use of tiny robots or nanorobots that can perform surgical procedures with high precision and efficiency [87]. These nanorobots can be guided to specific locations within the body using advanced imaging techniques, and they can then perform tasks such as delivering drugs, removing tumors, or repairing damaged tissues. Nano-bioelectric medicine, on the other hand, involves using electrical signals to stimulate the bodys healing processes [88,89]. This emerging field focuses on the use of nanoscale technologies to access and control the electrical activity of cells and tissues in order to treat a wide range of medical conditions, including chronic pain, wound healing, and heart disease [90]. Both surgical nanorobotics and nanobioelectric medicine have the potential to revolutionize the field of medicine and improve patient outcomes. However, there is still much research needed to fully explore the potential of these technologies and ensure their safety and efficacy [88].

Programming, engineering, and biological fields are working inter-connectively to develop a surgical nanorobot that works through the vascular system. These small-scale devices are manufactured with the multipurpose function of searching diagnostics and treatments against lesions and pathogens [87,88]. These robots work at a minute scale that can be used to cut even a single dendrite and neuron at the cellular surgery level without causing harm to other neurons bound in a complex network. These experiments have been confirmed in animal models where a nanoscissor action has been governed by these nanorobotics [91]. The results have pushed scientists to perform further experiments before optimizing surgical conditions on diseased patients. A new wave of bioelectric medicine is also in the market which adheres to biological components for more effective diagnostic and therapeutic therapies. This nanobioelectronic is being employed in cancerous diseases, cardiovascular disorders, and other malfunctions in the human body [92]. However, many improvements are needed to successfully apply this technology in a clinical setting for multipronged complex diseases.

Nanogenerators, as the name indicates, are a class of self-powered and implantable medical nanosensors. They work on the principle of conversion of mechanical energy from body movement into an electric spark [87]. As the body converts chemical energy from glucose, muscle converts this energy to mechanical energy and in turn these nanogenerators convert it into electric energy which can be used to charge and power implantable nanodevices that are aggressively being manufactured for medical purposes nowadays [88]. Implantable medical nanogenerators (IMNGs) are miniature devices that use mechanical energy from body movements to generate electrical energy [87]. They can be implanted inside the human body and used to power various medical devices, including pacemakers, neurostimulators, and drug delivery systems [93].

IMNGs are made up of thin layers of materials, such as piezoelectric materials, which convert mechanical energy into electrical energy. These materials generate electric charges when they experience mechanical stress, such as bending or pressure [87]. They can also be designed to harvest energy from other sources, such as temperature changes or fluids in the body [88]. IMNGs have several advantages over traditional batteries used to power implantable medical devices. They can eliminate the need for battery replacements, which can be invasive and costly. They can also improve device reliability as battery failures can cause serious medical problems [93]. Additionally, IMNGs are environmentally friendly since they do not require the disposal of toxic batteries [94].

Despite their potential benefits, there are still challenges to overcome in developing IMNGs. The devices must be durable enough to withstand the harsh conditions inside the body, including high temperatures and corrosion from body fluids [95]. They must also be small enough to be implanted inside the body without causing discomfort or obstruction [94,95]. Overall, IMNGs hold great promise for improving the safety, reliability, and convenience of implantable medical devices in the future. Therefore, researchers are continuously working toward their development to make them practical for human use.

Anesthesia induction is a critical step in dental surgeries and other sensitive medical procedures, such as brain surgeries. For such anesthesia induction procedures, researchers are working on nanorobotic suspension mixtures that make a colloidal suspension with millions of nanoscale active analgesic nanoparticles [96]. These nanoparticles work on patients gingival and other sensitive portions and penetrate deep up to the level of loose tissue. This passage of nanomaterials is conducted via the combinational principles of chemical and temperature gradients and positional navigation that are monitored and controlled by onsite nanocomputers [97]. This nanoscale anesthetic action helps to carry out the desired effect, attained quickly with an even distribution of anesthetic in the projected organ such as the dental surface. The sensitivity action can also be controlled for a particular tooth for which surgical action is required. After the completion of surgeries, nanorobots are controlled via nanocomputers to restore tooth sensitivity to normal [98].

Nanodentistry is a separate branch of nanomedicine that involves a broad range of applications of nanotechnology ranging from detection to diagnosis, to cure treatment options and prognostic details about tooth functions [99]. A wide spectrum of oral health-related issues can be dealt with using nanomaterials [100]. These nanomaterials derive their roots from tissue engineering and biotechnologically manufactured dental nanorobotics [100,101]. Some recent advances under oral nanotechnology may include treatment options such as anesthesia, dentition renaturalization, hypersensitivity cures, orthodontic realignment problems, and modernized enameling options for the maintenance of oral health [99,102].

The nanoscale technology used for such functions are named mechanical dentifrobots. They work to sensitize nerve impulse traffic at the core of the tooth in real-time calculation and hence could regulate the tooth tissue penetration and maintenance for normal functioning [103]. The functioning is coupled with programmed nanocomputers to execute actions from external stimuli via connection with the localized internal nerve stimuli. These mechanistic insights could help dental surgeons suggest a strategic treatment option that may be conducted directly via in vivo nanorobot action using acoustic signals, as elaborated earlier [100,101,102,103,104]. Some of the applications of nanotechnology in the field of dental science have been compiled at the end of this section in .

Major applications of nano-dentistry.

Scientists are further working to use nanotechnology for the creation of dental cures and treatment strategies. This may include the stimulation of the natural biomineralization process or the utilization of nanomaterials for artificial tooth development with sensitivity programed by nanorobotics [100,105]. They are trying to develop the hardest tissue enamel by using nanoscale manufacturing of nanorods derived from calcium hydroxyapatite crystals to help regulate the function of teeth. Additionally, reconstructive dental nanoparticles are utilized to offer patients a rapid and long-term cure against hypersensitivity [106].

Repositioning of the tooth is a matter of greater concern for patients as it sets the basis for further cure or disruption of dental health in case of maladjustment. Orthodontic nanorobots could be used in this case to manipulate tissues in such a way that a smooth painless straightening, rotation, and repositioning of the tooth could be attained [107]. Moreover, with time, customers are more interested in improving the aesthetic standing of their physical appearance, and so the concept of dental esthetics has emerged. In this regard, nanotechnology is considered to perform actions such as excavating dental amalgams or remanufacturing teeth alongside fillings, crowns, and other such modifications [107,108].

Much more effort is being put into securing dental durability and the appearance of teeth in normal dentistry practices. Nanotechnology provides a more secure and long-lasting solution in the form of nanostructured dental materials with carbon nanotubes that provide fracture-resistant properties [109]. Additionally, simpler dentifrobots are being incorporated into mouthwashes and toothpastes to replenish dental surfaces on a routine basis for cleaning and continuous calculus debridement [110]. These dentifrobots have the ability to highlight and destroy specific pathogenic bacteria from the mouth and retain the useful oral microflora in a healthy balance [111]. All these benefits delay the conventional causes and processes of dental decay with the remedial disappearance of oral diseases, especially in the early years [100,112].

In the world of medicine, complex and incurable diseases such as cancer are always given a special focus to find treatment and early diagnosis options for these modalities [113]. Nanotechnology is providing a good opportunity for researchers to develop such nano-agents, fluorescent materials, molecular diagnostics kits, and specific targeted drugs that may help to diagnose and cure disease in a better way in the future [114]. Scientists are trying various protocols to conjugate already available drugs with nanoparticles to enhance drug specificity and targeting in organs [113,114,115].

Nanomedicine acts as the carrier for hundreds of specific anticancerous molecules that could be projected at tumor sites. Moreover, the tumor imaging and immunotherapy approaches linked with nanomedicine must also be kept in mind when diving deep into nanomedicine and cancer links [34]. The effectiveness of nanomaterials in cancer therapies has pushed scientists to replace traditional cancer therapy approaches with targeted therapies that may be utilized alone or in conjugation with already available anti-cancerous drugs [16,34]. The focus is also being drawn toward lessening the impact of chemotherapeutic drugs by increasing their tumor-targeting efficiency and improving their pharmacokinetic and pharmacodynamic properties. Similarly, heat-induced ablation treatment against cancer cells alongside gene therapy protocols are also being coupled with nanorobotics [52].

Some other cancer treatment options, in the form of enhanced tissue imaging and tumor microenvironments, as well as adjustment by the release of nanoparticle-bounded drugs, are being practiced in the oncology field [59,116]. These nanomedicines hold the potential to overcome drug solubility, instability, and resistance issues. Various nanomedicines that act as anticancerous medicines are being researched, while some have been approved by the US Food and Drug Administration (FDA) and European Medicine Agency (EMA) [117]. These anticancerous drugs may utilize the Enhanced Permeation and Retention Effect (EPR effect) and/or active targeting of nano assemblies such as liposomes, albumin nanospheres, micelles, and gold nanoparticles [118]. Some of the applications of nanotechnology in the oncology field are discussed in the following section and a summary () is shown at the end of this section.

Applications of Nanotechnology in Oncology field.

Cancer diagnosis is the most observable problem in cancer patients. Cancer largely remains uncured due to late detection in the third or fourth stages. To fight this cause, nanotechnology is being employed to allow early detection of tumors in organs [16]. Nanotechnology provides a very sensitive and specific multiplexed measurement capacity to detect cancer biomarkers in extracellular settings and in vivo bioimaging techniques [19]. Nanotechnology has enormous potential in the field of cancer diagnosis. Nanoparticles are incredibly small and can penetrate cell walls and the bloodbrain barrier. This makes them ideal for delivering drugs and other therapeutic agents to cancer cells. They can also be used to detect cancer cells and identify the location and nature of the disease [119].

One of the most promising areas of nanotechnology in cancer diagnosis is the development of targeted nanoparticles. These are nanoparticles designed to adhere specifically to cancer cells, allowing them to be easily identified and targeted by doctors. This could result in more accurate early detection, better monitoring of cancer progression, and faster diagnosis [120]. Another promising application of nanotechnology in cancer diagnosis is in the development of biosensors. Biosensors are small devices that can detect specific biomarkers in a patients blood or other bodily fluids. These biomarkers can be indicative of cancer and could be used to detect cancer at an early stage [92,93].

In conclusion, nanotechnology has enormous potential in the field of cancer diagnosis. With targeted nanoparticles and biosensors, it could help in the development of a more accurate, non-invasive and effective way to diagnose cancer. However, the challenges pertaining to such diagnostic kits remain and the need is to overcome these challenges and update the nanotechnology-based diagnostic methods for cancer and other disease diagnostics and prognoses in the future [19,120].

Multifunctional theranostics therapy is an emerging field in cancer treatment that combines multiple modalities into a single treatment approach. This approach aims to both diagnose and treat cancer using nanomaterials. Nanomaterials, such as nanoparticles, are highly versatile due to their unique properties at the nanoscale [121]. They can be engineered to have various functionalities, such as imaging capabilities, drug delivery systems, and targeted therapy agents. By using these multifunctional nanomaterials, theranostics therapy can provide simultaneous cancer diagnosis and treatment [122]. In parallel, the term multimodal refers to the combination of multiple treatment modalities in a single therapy [123]. In the context of theranostics therapy, multimodal treatment can involve different approaches, such as chemotherapy, radiotherapy, and immunotherapy [124]. These modalities can be incorporated into nanomaterials used for therapy, allowing for targeted delivery and enhanced efficacy. The theranostic approach also enables real-time monitoring of treatment outcomes [124]. By incorporating imaging agents into nanomaterials, clinicians can track the distribution and effectiveness of the therapy. This information helps guide treatment decisions and allows for adjustments to optimize patient outcomes [123]. Thus, the combination of multifunctional and multimodal theranostics therapy using nanomaterials holds great promise in the fight against cancer. It offers the potential for personalized and targeted treatment, improved efficacy, and reduced side effects compared to traditional cancer therapies [121,125].

Targeted nano drug delivery technology for cancer therapy is a form of treatment that uses nano-sized particles to deliver drugs specifically to cancer cells in the body. These nanoparticles can be engineered to selectively bind to cancer cells, allowing the drugs to be delivered directly to the tumor site, while minimizing damage to healthy tissues [5]. The development of targeted nano drug delivery systems has several advantages in cancer therapy. Firstly, it can enhance the efficacy of the drugs by increasing their concentration at tumor sites. This is especially important for drugs with low solubility or high toxicity as it allows for higher doses to be delivered directly to the cancer cells [24]. Additionally, targeted nano drug delivery systems can help overcome some limitations of traditional chemotherapy, such as poor drug bioavailability or resistance. By encapsulating the drugs within nanoparticles, their stability and solubility can be improved, leading to better drug delivery and higher therapeutic effects [5,126].

There are various types of targeted nano drug delivery systems being explored, including liposomes, polymeric nanoparticles, dendrimers, and carbon nanotubes. These nanoparticles can be functionalized with ligands or antibodies that specifically bind to receptors or proteins overexpressed on the surface of cancer cells [127]. This targeting moiety allows for the selective binding and internalization of nanoparticles into cancer cells, enabling efficient drug delivery. Furthermore, targeted nano drug delivery systems can also be combined with imaging agents, enabling real-time monitoring of drug distribution, tumor targeting, and uptake [24,128]. This helps in tracking the therapeutic response and adjustment of treatment protocols as needed [128]. Overall, targeted nano drug delivery technology has the potential to revolutionize cancer therapy by improving the efficacy and safety of drugs, minimizing systemic side effects, and enabling personalized medicine approaches. However, further research and development is still needed to optimize these systems and ensure their clinical translation [128,129].

Nanotechnology and magnetic drug delivery technology are both innovative approaches in the field of medicine that improve drug delivery and enhance treatment effectiveness. Magnetic drug delivery technology utilizes the application of an external magnetic field to guide drug-loaded nanoparticles to a specific site within the body [130]. Magnetic nanoparticles can be functionalized with drugs and then injected into the bloodstream. By applying a magnetic field externally, the nanoparticles can be directed toward the desired location, such as a tumor [131]. This approach allows for more precise drug delivery, minimizing systemic exposure and reducing side effects [39]. Similarly, by engineering nanoparticles, researchers can create drug carriers with unique properties that are not in conventional drug delivery systems [130,131,132]. These nanoparticles can be functionalized and designed specifically to target diseased cells or tissues, improving drug concentration at the desired site and minimizing off-target effects [131].

Additionally, nanoparticles can protect the drug payload from degradation, resulting in improved stability and prolonged drug release. Thus, the combination of nanotechnology and magnetic drug delivery technology has shown promise in several areas of medicine [130]. For example, in cancer treatment, magnetic nanoparticles can be used to deliver chemotherapy drugs directly to tumors, increasing drug concentration at the tumor site and reducing toxicity in healthy tissues. This approach can enhance treatment efficacy while minimizing adverse effects [133]. Furthermore, magnetic drug delivery can also be utilized in targeted therapy for other diseases, such as neurological disorders. Nanoparticles loaded with neuroactive drugs can be guided to specific regions in the brain using externally applied magnetic fields, allowing for more targeted treatment and potential reduction in systemic side effects [133]. Thus, the integration of nanotechnology and magnetic drug delivery technology has the potential to revolutionize drug delivery by improving targeting, reducing side effects, and enhancing treatment outcomes. Ongoing research and development in this field hold great promise for the future of medicine.

As nanotechnology is making progress in the field of medicine and biological sciences, eyes are on the board as to how this technology will bring revolution to medical machinery [25]. It is predicted that soon, micro and nanoscale materials will be integrated with useful robotic characteristics that may include nanoscale manipulator arms, sorting rotors, reagent purification kits, and super diagnostic surfaces that will be modeled to respond to particular disease diagnostics and treatment. These nanomaterials and robotic connections are assumed to be controlled via nanocomputers [25,134].

Nanocomputers are expected to control, activate, deactivate, and deter the response rates of nanomechanical devices [134]. They will be programed to execute specified medical and dental operations with a connection to a wider network of interconnected nanocomputers, such as programmed nanomachines and robotics, which have the potential to allow physicians and clinicians to perform precise medical procedures at a subcellular level [135,136,137]. Furthermore, these robotic elements are expected to work in gerontological and pharmaceutical research phases, diagnostics, and dentistry [138].

In addition to the application of nanomedicine to humans, beneficial applications of nanomedicine are now being used on animals. Multiple variations of nanovaccines and nanoadjuvants have started their way into veterinary sciences [11,139]. The previously used animals therapeutic, diagnostic, treatment, and veterinary vaccinations along with disinfection, breeding, reproduction, and nutritional concerns are now being modernized using the concept of nanotechnology [139].

Nanotechnology has the potential to revolutionize the field of veterinary medicine, offering new diagnostic tools and treatment options for animals. In the area of diagnostics, nanotechnology can improve the accuracy and sensitivity of diagnostic tests used to detect various diseases [140]. Nanoparticles can be engineered to bind to specific biomarkers in the body that are indicative of disease, allowing for early detection and treatment [140]. In the field of therapeutics, nanotechnology can improve drug delivery systems, enhancing drug efficacy while minimizing side effects. Nanoparticles can be designed to improve drug solubility, stability, and specificity, ensuring that drugs reach their intended targets and remain active for longer periods of time [139,140,141,142].

Additionally, nanotechnology can be used to develop novel vaccines and immunotherapies, as well as new tools for regenerative medicine. For instance, nanoparticles can be used to create scaffolds for tissue engineering and repair, promoting the growth of new tissue and accelerating healing processes [73,85,86]. The use of such small-scale nanomedicine shows a direct impact on public health due to the interconnectedness among humans and animals within the same living environment. The effort is going on to increase meat and milk production, leading to a reduction in vaccine residues and drug resistance problems in veterinary medicine [142,143]. Moreover, this medicinal revolution remains cost-effective and helps to minimize the amount of discarded milk and meat products. In addition to that, in modern pet care, nutritional and hygienic products are also being introduced in the market under the genesis of successful practices in nanotechnology [143]. Overall, nanotechnology offers exciting possibilities for improving animal health and welfare and has the potential to revolutionize veterinary medicine.

Nanosensors refer to small devices that can detect and analyze chemical or biological agents at the molecular level. They have various applications, including monitoring air quality and detecting pathogens in food and water [12]. Nano-microbivors, on the other hand, are small (microscopic) organisms that can consume or break down contaminants such as organic chemicals and heavy metals in the environment [17]. They can be used for bioremediation purposes and for treating contaminated soil and groundwater [144,145]. There is an interlink between these concepts, in that nanosensors and nano-microbivors can be used in the detection and remediation of chemical warfare agents [146]. For example, nanosensors can be developed to detect the presence of chemical warfare agents in air or water, while nano-microbivors can be used to break down or detoxify these agents in the environment [146,147]. In this way, these technologies are important tools in ensuring national and global security.

A new wave of nanosensors is being developed to be utilized for military purposes against detection of airborne and released chemical agents that could be easily exhaled and inhaled with toxic outcomes [12,17]. Phagocytes have a cellular clearing digestive function; based on this principle, artificially designed nanoscale microbiomes are being used in studies to clean the bloodstream by digesting toxic pathogens [146]. They perform this function in a very limited time as compared to other medication options without causing any toxicity or septic shock conditions. A similar principle of action will be utilized to detect the amount of inhaled prohibited drugs such as marijuana, banned substances, and alcohol concentrations in individuals, against which the use of such substances is strictly prohibited in patients [148]. Such advanced technologies may take the place of traditional procedures, which are extensive and time-consuming diagnostic procedures.

During the COVID-19 pandemic, nanomedicine has played a crucial role in developing diagnostic tools, treatment strategies, and vaccine delivery methods. The link between the coronavirus and nanoparticles based on size and function is relatively straightforward. In terms of size, both the virus particles and nanoparticles are tiny particles with a size on the nanoscale [149]. This small size allows them to interact with each other on a very tiny scale. Similarly, in terms of functional similarities, nanoparticles can be engineered or designed to have specific functions. For example, some nanoparticles can be coated with molecules that make them stick to viruses such as the coronavirus [150]. This function is essential because it allows nanoparticles to grab onto the virus. Thus, in the context of the coronavirus, scientists have explored how nanoparticles can be used in various ways including detection, treatment, and protective responses. Nanoparticles can be designed to bind to specific parts of the coronavirus. When they attach to the virus, they can change color or emit light, making it easier for scientists and doctors to detect the presence of the virus in a sample, such as a patients blood or saliva [151]. Similarly, nanoparticles can also be used to deliver medicines directly to the virus or infected cells. Think of nanoparticles as tiny delivery vehicles that can carry antiviral drugs right to the site of infection, potentially making treatments more effective [152]. In addition, regarding the protective technologies against COVID-19, Some masks and face coverings have been designed with nanoparticle coatings that can trap and neutralize viruses, including the coronavirus, when they come into contact with the masks surface [149,151]. Furthermore, nanoparticles have been used to create highly sensitive and specific diagnostic tests that can detect SARS-CoV-2 in patient samples [149]. Nanoparticles have also been used to develop therapeutics that can directly target the virus, as well as improve the delivery and efficacy of existing drugs [149].

In addition, nanotechnology has been used to improve the stability and efficacy of vaccines, as well as develop new delivery methods such as nasal sprays and microneedle patches [149,150]. These approaches can help increase vaccine accessibility and effectiveness, particularly in resource-limited settings. The breakthrough and rapid responses coming from nanomedicine can be ascertained by the fact that nanotechnology is also being utilized for vaccine drug manufacturing technologies against COVID-19 [151]. Since nanomedicine has already proven its benefits for disease diagnosis, treatment, and prevention, it is being employed to tackle the pandemic. Now, nano-based technology is on hand and is being considered for utilization in manufacturing antiviral technology to integrate into personalized medical equipment and to manufacture nano-based drugs [150,151]. The sole purpose is the greater safety of medical workers and to save patients suffering from the impediments of the coronavirus with more sensitive medicine and machinery.

In this regard nanomaterials, such as quantum dots, are being introduced into biosensors for diagnostics experiments and other nanoassemblies, such as liposomes, polymeric and lipid nanoparticles, metallic nanoparticles, and micelles, which are being utilized for antiviral drug encapsulation and drug conjugation [150,151,152,153]. The great benefit would be increased pharmacological impact and more efficient drug targeting. Studies are showing that these antiviral properties of nanoparticles function by blocking the binding, entry, and replication of coronavirus in the body [154]. With this technology, the toxicity linked to normal body cells owing to nanoparticle application is the major factor of concern and thus needs to be investigated and improved for future applications [155]. Overall, nanomedicine holds great promise in the fight against COVID-19 and could potentially revolutionize the way we diagnose, treat, and prevent infectious diseases in the future. below shows the link between nanoparticles and coronavirus in terms of the chemistry of the structure, size, and functionality that could be used as an exemplary overview as to how nanotechnology could be majorly utilized to discover antiviral treatments in the future. Commercial applications of nanotechnology in medical field are summarized in .

A link between coronavirus and nanoparticles based on size and function.

Examples of nanotechnological applications and their commercialization in the medical field.

The side effects of nanotechnology are of great concern for humans, animals, and the overall environment. While the toxicity attached to these assemblies is poorly understood, the scientific community remains unsure as to what level they can extend the applications of nanotechnology, especially in medicine, which is quite a sensitive domain of healthcare [142]. In previous years, some nano-based products were introduced but later pulled back from the market owing to the reported side effects in the general public. The risk assessment of nanomedicine is thus a critical topic and needs to be assessed soon [145].

The need is to prioritize experiments for nanoparticle safety, dosing adjustment, and usage. The miracles of nanotechnology itself can be used in sensors and markers for biological, chemical, and environmental remediations [162]. Toxicity profiling of consumer products should be specifically carried out. Skin care and dental products containing different nanomaterial liposomes, cubosomes, solid lipid nanoparticles, and dendrimers must be specifically assessed, and their side effects must be determined so that more modified, effective, and harmless nanoemulsions can be introduced and utilized in the future [163].

Similarly, the issue of bioaccumulation and persistence is attached to nanotechnology. Nanomaterials have the potential to persist in the environment for extended periods and accumulate in living organisms [162,163,164]. This can lead to potentially adverse effects on both human health and ecosystems. Additionally, in healthcare settings, medical professionals who handle nanomaterials may be at risk of potential exposure through inhalation, dermal contact, or ingestion. Safe handling practices and adequate protective measures must be implemented to minimize exposure risks [165]. Moreover, the use of nanomaterials in medical applications also raises ethical considerations regarding informed consent, privacy, equity of access, and potential impacts on vulnerable populations. There is a need to address these ethical concerns to ensure the fair and responsible use of nanomaterials in healthcare [166].

To ensure the safe and sustainable use of nanomaterials in the medical field, several measures can be implemented, such as rigorous and comprehensive risk assessments, which should be conducted to evaluate the potential hazards and risks associated with specific nanomaterials before their deployment in medical applications. Similarly, adequate regulatory frameworks should be in place to ensure the safe production, handling, and utilization of nanomaterials [142,166]. This includes the evaluation of their safety, labeling requirements, and monitoring of their effects in healthcare settings. Additionally, standardized testing methods should be developed to assess the safety and efficacy of nanomaterials for medical use. This includes standardized protocols for toxicity testing, characterization, and quality control. Furthermore, strict control measures should be implemented to minimize occupational exposure to nanomaterials [165]. This includes the use of engineering controls, personal protective equipment, and employee training programs. Moreover, transparent communication about the potential risks and benefits associated with nanomaterials is essential for establishing trust among stakeholders, including healthcare professionals, patients, and the general public [165,166].

The need is to prioritize experiments for nanoparticle safety, dosing adjustment, and usage. The miracles of nanotechnology itself can be used to produce sensors and markers for biological, chemical, and environmental remediations [166]. Toxicity profiling of consumer products should be specifically carried out. Skin care and dental products containing different nonmaterial liposomes, cubosomes, solid lipid nanoparticles, and dendrimers must be specifically assessed, and their side effects must be determined so that more modified, effective, and harmless nano-emulsions can be introduced and utilized in the future [145,166]. Overall, by evaluating potential risks, implementing appropriate regulatory measures, and promoting responsible use, nanomaterials can be safely and sustainably utilized in the medical field for improved diagnostics, drug delivery, and disease treatment.

Nanomaterials hold significant promise for various biomedical advancements and industrial applications. However, their unique physicochemical properties raise concerns about their potential impact on human health and the environment. In order for medical nanomaterials to enter the market, there are many obstacles to overcome, such as FDA certifications and permits, as well as safety and ethical concerns. In recent years, regulatory bodies worldwide have focused on developing appropriate frameworks to ensure the safe and responsible use of nanomaterials. Such an issue should be addressed more intensively in the coming years of nanotech research. Review papers, in this regard, should aim to provide researchers, policymakers, and industry professionals with a comprehensive understanding of the recent regulatory affairs surrounding nanomaterials. By critically examining the current state of nanomaterial regulation, this paper highlights the need for harmonization and collaboration among regulatory agencies worldwide. Regulating industrialization affairs surrounding nanomaterials in medical sciences involves several steps. It is important to note that these steps provide a general framework, but the specific details and processes may vary depending on the jurisdiction and specific requirements of each country or region. A general outline of the process is provided in a table format () below. Steps needed to regulate the industrial affairs of nanotechnology are shown in .

FDA approved and commercialized nanomedicines.

Steps needed to regulate industrialization affairs surrounding nanomaterials in the medical sciences.

A comprehensive search strategy was adopted for this systematic review to include data from diverse, recent, and the most cited sources of study.

Data were collected via a systematic literature search through various online sources including Google Scholar, PubMed, NIH (National Library of Medicine), Web of Science, European database, Springer, and Embase databases. Since the study was focused on the applications of nanotechnology in medicine and healthcare, the major research items were nanotechnology, nanobiotechnology, nanomedicine, nanotechnology and medical applications, nanotechnology and diagnosis, nanotechnology and treatment, nanotechnology and drug-delivery, and nanotechnology and healthcare and esthetics, among other similar search terms. After a thorough analysis of titles and abstracts of publications related to applications of nanotechnology in the medical and healthcare industry, the data was selected to be part of this study. Only studies published in the English language were included in this study. Moreover, only data from 2010 onwards were included in the article.

Multiple types of sources were used, including data from research articles, book chapters, review articles, case reports, clinical trials, and case studies published starting beginning in 2010. Studies with incomplete citations and published before 2010 were excluded from the study.

The future of nanotechnology in healthcare and medicine holds immense potential for revolutionizing the way we diagnose, treat, and prevent diseases. Nanotechnology involves the manipulation of materials at such a small scale where the properties of materials significantly differ from their bulk counterparts, allowing for precise control of their physical, chemical, and biological properties. This opens up new opportunities for developing novel therapies, targeted drug delivery systems, and sensitive diagnostic tools. In addition to drug delivery, targeted delivery, improved drugs, limited dosages, and reduced systematic side effects, nanoparticles can also be used to enhance the efficacy of existing drugs by improving their solubility, stability, and bioavailability. Additionally, nanotechnology-based sensors and devices can monitor patient health in real-time, enabling early detection and personalized treatment plans. In the future, nanotechnology may even enable the development of nanorobots that can navigate through the bloodstream to target and destroy cancer cells or deliver payloads of drugs to particular tissues.

The broad spectrum of nanomedicine covered in this article may be lacking in various other aspects of nanomedicine still in the research pipeline. The vision of nanotechnology might seem heretic and abstract, similar to the in silico experimentation and computational bioinformatics field that was criticized a few years back. However, the field of nanobiotechnology is rapidly appearing as a cutting-edge technology of the 21st century, with diverse implications in science and technology. The theoretical knowledge is there, and applied research is ongoing to make it more progressive. It is predicted that soon, nanotechnology will not remain an option but rather be compulsory in the medical industry. As soon as the cost associated with technology becomes accessible, it is predicted to affect our dentistry, healthcare, and human life more profoundly than in the past. The major need is to curtail the toxicological concerns and risks that are attached to high doses and the excessive use of nanomaterials in drug and treatment regimes. This is important if scientists want to enable the successful operation of nanotechnology in medicine. Overall, the future of nanotechnology in healthcare and medicine holds great promise for improving patient outcomes and revolutionizing the way we approach disease prevention and treatment.

Conceptualization, S.M., K.M. and Y.W.; methodology, S.M., K.M. and Y.W.; validation, S.M., K.M. and Y.W.; formal analysis, S.M., K.M. and Y.W.; resources, K.M. and Y.W.; data curation, S.M., K.M. and Y.W.; writingoriginal draft preparation, S.M., K.M. and Y.W.; writingreview and editing, S.M., K.M. and Y.W.; supervision, Y.W.; funding acquisition, K.M. All authors have read and agreed to the published version of the manuscript.

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Emerging Applications of Nanotechnology in Healthcare and Medicine

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Magellan Stem Cells welcomes $7 million federal government grant

Saturday, September 21st, 2024

Magellan Stem Cells has welcomed a $7 million grant from the federal governments Medical Research Future Fund (MRFF) to help fund a Phase 3 human trial of the companys donor stem cell treatment for osteoarthritis.

In announcing the grant, Health Minister Mark Butler said, We are living through a supercharged period of discovery in health and medical research, and the Albanese Government is proud to support Australias world class researchers.

Stem cell therapies could provide innovative treatments for many chronic and inherited diseases we cant yet treat effectively. Such therapies could also revolutionise how we test and develop new medications."

Magellans Phase 3 osteoarthritis trial is one of six projects sharing $34.5 million supported by the MRFF Stem Cell Therapies Research Grant Opportunity.

Osteoarthritis is a huge cause of pain and disability in Australia, said Minister Butler. Up until now the treatments only extended to pain relief and potentially replacement surgery, like knee replacements.

The trial of Magellans donor stem cell treatment for osteoarthritis is scheduled to begin next year.

The grant was announced following the publication of research by Magellan, which demonstrated the potential for significant therapeutic benefits of its MAG200, an off-the-shelf donor stem cell therapy for osteoarthritis.

The research findings are published in Osteoarthritis and Cartilage Open.

Lead researcher and Magellan chief medical officer, Associate Professor Julien Freitag, said, We are very grateful to the Australian Government and the

Medical Research Future Fund (MRFF) for their support for this potentially life-changing technology.

The grant is a vote of confidence in the future of the Australian biotech sector, stem cell technology and Magellans ground-breaking research.

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Magellan Stem Cells welcomes $7 million federal government grant

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Nxera’s Partner Cancer Research UK to Present on Phase 1/2a Clinical Trial with Cancer Immunotherapy Drug HTL0039732 at ESMO

Friday, September 13th, 2024

Tokyo, Japan and Cambridge and London, UK, 13 September 2024 – Nxera Pharma (TSE: 4565, “Nxera”) and Cancer Research UK announce an upcoming presentation on the ongoing Phase 1/2a clinical trial (NCT05944237) of Nxera’s immunotherapy drug HTL0039732 (also known as NXE0039732) at the European Society for Medical Oncology Congress (ESMO) 2024, taking place on 13–17 September in Barcelona, Spain.

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Nxera’s Partner Cancer Research UK to Present on Phase 1/2a Clinical Trial with Cancer Immunotherapy Drug HTL0039732 at ESMO

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Top preventive health tips your internist wants you to know – American Medical Association

Friday, September 13th, 2024

Top preventive health tips your internist wants you to know  American Medical Association

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Top preventive health tips your internist wants you to know - American Medical Association

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The Progression of Regenerative Medicine and its Impact on Therapy …

Friday, September 13th, 2024

Clin Transl Sci. 2020 May; 13(3): 440450.

1Division of Cardiac Surgery, University of Ottawa Heart Institute, OttawaOntario, Canada

2School of Human Kinetics, University of Ottawa, OttawaCanada

1Division of Cardiac Surgery, University of Ottawa Heart Institute, OttawaOntario, Canada

3Department of Cellular & Molecular Medicine, University of Ottawa, OttawaCanada

1Division of Cardiac Surgery, University of Ottawa Heart Institute, OttawaOntario, Canada

2School of Human Kinetics, University of Ottawa, OttawaCanada

3Department of Cellular & Molecular Medicine, University of Ottawa, OttawaCanada

Received 2019 Nov 6; Accepted 2019 Nov 7.

Despite regenerative medicine (RM) being one of the hottest topics in biotechnology for the past 3decades, it is generally acknowledged that the fields performance at the bedside has been somewhat disappointing. This may be linked to the novelty of these technologies and their disruptive nature, which has brought an increasing level of complexity to translation. Therefore, we look at how the historical development of the RM field has changed the translational strategy. Specifically, we explore how the pursuit of such novel regenerative therapies has changed the way experts aim to translate their ideas into clinical applications, and then identify areas that need to be corrected or reinforced in order for these therapies to eventually be incorporated into the standardofcare. This is then linked to a discussion of the preclinical and postclinical challenges remaining today, which offer insights that can contribute to the future progression of RM.

In 1954, Dr. Joseph Murray performed the first transplant in a human when he transferred a kidney from one identical twin to another.1 This successful procedure, which would go on to have a profound impact on medical history, was the culmination of >50years of transplantation and grafting research. In the following years, organ replacement became more widespread but also led to a plateau in terms of landmark successes.1 The technology was working, but limitations were already being encountered; the most prominent of them being the lack of organ availability and the increasing need from the aging population.2 During the same time period, chronic diseases were on the rise and the associated process of tissue degeneration was becoming evident. Additionally, the available clinical interventions were merely capable of treating symptoms, rather than curing the disease, and, therefore, once a loss of tissue function occurred, it was nearly impossible to regain.3 Overall, the coupling of all these factors that took place in the 1960s and 1970s created urgency for disruptive technologies and led to the creation of tissue engineering (TE).

TE can be described as a field that applies the principles of engineering and life sciences toward the development of biological substitutes that restore, maintain, or improve tissue function or a whole organ.4 TE is considered to be under the umbrella of regenerative medicine (RM) and, according to Dr. Heather Greenwood et al., regenerative medicine is an emerging interdisciplinary field of research and clinical applications focused on the repair, replacement or regeneration of cells, tissues or organs to restore impaired function resulting from any cause, including congenital defects, diseases, trauma and aging.5 It uses a combination of technological approaches that moves it beyond traditional transplantation and replacement therapies. These approaches may include, but are not limited to, the use of soluble molecules, gene therapy, stem cell transplantation, tissue engineering, and the reprogramming of cell and tissue types.3, 6, 7 A summary of the recent history of RM is presented in Figure.

A summary timeline of the recent history of regenerative medicine (RM). Selected milestones in the development of RM are presented starting from the 1950s all the way up to the present day.

Although RM may have seemed novel, the principles of regeneration are as old as humanity and are found in its many cultures.8 A common example used is the tale of Prometheus that appeared in 8th century BCE. Prometheus, an immortal Titan in Greek mythology, stole fire and gave it to humanity for them to use, defying the gods in consequence. As punishment, Zeus decreed that he was to be bound to a rock where an eagle would feast on his liver every day and said liver would regenerate itself every night, leading to a continuous loop of torture.9 RM came about at the time it did, not only because of the combining factors mentioned above, but also because researchers had been successfully keeping tissue alive in vitro and understanding the biological processes involved in regeneration and degeneration. Consequently, possible therapeutic outcomes came into fruition. Since the arrival of TE and RM, strides made on the benchside have been ever increasing with now >280,000 search results on PubMed relating to regeneration. Discoveries and advances made by cell/molecular biologists, engineers, clinicians, and many more led to a paradigm shift from treatmentbased to curebased therapies.10 In addition to Greenwoods definition, RMs arsenal now contains controlled release matrices, scaffolds, and bioreactors.5, 8 Despite this impressive profile on the benchside, RM has so far underperformed in terms of clinical applications (i.e., poor therapy translation).8 Simply put, a disappointing number of discoveries are making it through clinical trials and onto the market.11 Although some experts say that the field is reaching a critical mass in terms of potential therapies and that we will soon see results, others, like Dr. Harper Jr. from the Mayo Clinic in Minnesota, say that the transformative power of RM is well recognized, but the complexity of translating isnt.7, 8, 12

This brings us to the subject matter of the present paper: RM and translation. The goals of this historical review are twofold. The first is to understand how RM, over the past 50years or so, has changed the way discoveries/new technologies are transferred to the clinic. How has the translational strategy changed in response to these new therapies? The second is to identify challenges that have led to RMs modest performance on the bedside. Some articles have already documented these but have focused on the clinical and postclinical factors, and whereas they will be briefly discussed here, the focus will be on preclinical factors.13 To accomplish these objectives, we will begin by summarizing the historical development of RM (which has been extensively documented by other works2, 3, 14, 15), followed by a detailed look at the definition of translational medicine (TM). With this background information established, we then look at the various preclinical and clinical impacts of RM on TM, as well as some of its effects on the private sector. Limiting factors of the field are then described, again focusing on those that are preclinical. This endeavor was initiated via a librarianassisted literature search for original research and historical documentation of the field of RM and other related subjects. The documents were then screened for relevance and the analyzed information was categorized into the themes discussed below. Conclusions were then drawn based on the interplay among these themes.

As mentioned, the idea of regeneration first started in myths and legends. This is logical because, as Drs. Himanshu Kaul and Yiannis Ventikos put it, myths shape ideas, and ideas then shape technologies.8 In addition to the tale of Prometheus, there are many others. For example, there is the Hindu myth of Raktabeej whose blood drops could each form a clone of himself, or the Indian story of the birth of the Kaurava brothers where pieces of flesh were grown in pots and treated with herbs to grow fullsized humans.8 The idea of regeneration has persisted throughout history and started to become a possibility in the early 1900s when scientists like Alexis Carrel (who invented the technique of cell culture) were finally able to keep cells and tissues alive outside of the body. This allowed them to study the mechanisms of cell renewal, regulation, and repair.8 In addition, studying regeneration goes handinhand with developmental biology. Seminal work in experimental embryology began in the 1820s with the detailed description of the differentiation of embryonic germ layers.16 An increased understanding of basic embryological mechanisms led to Hans Spemanns Nobel Prize for his theory of embryonic induction; a field that was further elaborated by his students and others, advancing it toward the possibility of cloning and demonstrating how development and regeneration are intimately linked.16 Before this era, the study of regeneration was done through the study of animals, with scientists studying the phenomena in serpents, snails, and crustaceans, for example.17, 18 However, the modern study of regeneration is said to have started with Abraham Trembleys study of the hydra, which showed that it was possible for an entire organism to regenerate from its cut appendage.19 The 18th century on through to the 19th century is also when scientists became intrigued by the amphibian newts and axolotls for their astonishing regenerative capabilities, which are still used today as the gold standard models for studying regeneration along with certain fish, such as the zebrafish.20

Now, although the term RM as we know it today would only be coined in 1999 by William Haseltine, the field itself started in the late 1970s in the form of TE (pioneered by Drs. Joseph Vacanti and Robert Langer) in the city of Boston.2, 14, 21 To address the need for novel therapies, biomedical engineers, material scientists, and biologists at Harvard and MIT started working on regenerating parts of the largest and simplest organ of the human body: the skin. In 1979, the first cellbased TE product appeared and was named Epicel.15 Developed by Dr. Howard Green et al., this technology consisted of isolating keratinocytes from a skin biopsy and having them proliferate outside of the body to make cell sheets that were then used as an autologous treatment for burn patients.15 Another famous product (this time allogeneic), developed in 1981, was Apligraf, a composite skin invention capable of rebuilding both the dermis and epidermis of skin wounds.15 With these two therapies and many more being created, TE in the 1980s was booming. At the time, researchers were also developing therapies for cartilage regeneration.

Once the 1990s came around, TE strategies were combined with stem cells (which had just been discovered) to create RM.3, 8 At that time, RM was a hot topic. After the first products for skin were commercialized, scientists became more enthused and started trying other tissues.15 Startup companies were popping up left and right, private funding was abnormally high, and public hype was gaining lots of traction. However, governments were not so quick to fund this research and took their time before making decisions, whereas private investors saw this field as very promising and thought it was their ticket to the top.14 Given that 90% of the funding of RM came from the private sector, this greatly influenced the direction of the research and its timeframe.14 People were simply trying to copy tissue formation rather than understanding it, so as to make the development process quicker.3 As a result, many of the technologies that initially looked promising failed in clinical trials or on the market.

These disappointing results coupled with the dot.com crash meant that by the end of 2002, the capital value of the industry was reduced by 90%, the workforce by 80%, and out of the 20 US Food and Drug Administration (FDA) products with clinical trials, only 4 were approved and none had any success.22 This phenomenon has been extensively studied and, according to Lysaght and Hazlehurst, five factors contributed to the industry crash22:

The products were not much better than the existing treatment options and so making the switch was not worth it for clinicians.

Even if the science was good, lowcost manufacturing procedures did not exist.

The approval process for these novel therapies was unrealistically challenging and the regulatory cost was too high.

Companies lacked the skill to market their new products.

The reimbursement strategies were unclear.

Despite these events, the industry had 89 firms survive the crash and stem cell research was not affected. In fact, from 2000 to 2004, the number of companies increased but the number of jobs decreased, which means investors were supporting research in basic and applied science with smaller firms that were lower risk, and by 2004, the field was dominated by startup companies.22 Before the crash, RM was primarily happening in the United States, but in 2004, other countries like the United Kingdom and Japan started catching up.22 The industry slowly started growing again. In 2006, the first engineered tissue (bladder) was implanted, and by 2008, commercial successes were being achieved.3, 10 As an example, hematopoietic stem cell transplants were approved and are now a curative treatment for blood disorders and other immunodeficiencies.7 Now, the RM field had ironically regenerated itself.3 It has gained increased governmental attention (federal funding has increased) and has been recognized as being at the forefront of health care.7, 22 There is once again intense media coverage that is raising public expectations.23 The number and variety of clinical trials is also increasing everywhere.23 According to allied market research, RM is predicted to be worth US $67.5 billion by 2020.10

Unfortunately, regardless of these seemingly cheerful notes, the fact remains that cell therapies remain experimental, except for the aforementioned hematopoietic stem cell treatments.13 The market for RM is still small and will remain so until RM proves that its therapies are better and cheaper than the existing ones.15 Yet, the pressure for clinical translation is increasing through the needs of the population, investors that are eager to make a return on their investments, and scientists who believe that these technologies are the future.23 Moreover, there has been a growing appreciation of the magnitude and complexity of the obstacles the field is facing, but it remains to be seen how they will be solved; although initial steps have already been taken, which will be discussed further below.

Now that we have established the background for RM, there needs to be a proper understanding of TM before conclusions on how the two are related can be drawn, which is the purpose of the following section.

The European Society for Translational Medicine (EUSTM) has defined TM as an interdisciplinary branch of the biomedical field supported by three main pillars: benchside, bedside, and community. The goals of TM are to combine disciplines, resources, expertise, and techniques within these pillars to promote enhancements in prevention, diagnosis, and therapies.24 TMs goals can be split into two categories: T1 and T2. T1 is to apply research from bench to bedside and back, whereas T2 is to help move successful new therapies from a research context to an everyday clinical context.25 In other words, TM is a medical practice explicitly devoted to helping basic research attain clinical application. Conceptual medical research, preclinical studies, clinical trials, and implementation of research findings are all included within TM.26

Between basic science and the clinic is an area that is popularly referred to as the valley of death.25 This gap is fraught with not only scientific obstacles (like an unknown molecular mechanism), but social and economic ones as well. This is where many novel ideas die and, consequently, companies are weary of going through this valley for fear of wasted financial resources.25 For these reasons, many of the approved drugs we get now are derivatives of others that have been previously approved.25 This is the area that TM seeks to impact, to be the bridge between idea and cure, and to act as a catalyst to increase the efficiency between laboratory and clinic.25, 26 The term bench to bedside and back is commonly used. The cost of development for a therapy is very high (estimated at US $800 million to $2.6 billion for a drug) because of increasing regulatory demands and the complexity of clinical trials, among others. TM aims to streamline the early development stages to reduce the time and cost of development.24

What will be important to note for the discussion below is that TM focuses more on the pathophysiological mechanisms of a disease and/or treatment and favors a more trialanderror method rather than an evidencebased method. Dr. Miriam Solomon argues in his book chapter entitled What is Translational Medicine? that most medical innovations proceed unpredictably with interdisciplinary teams and with shifts from laboratory to patient and back again, and that freedom of trialanderror is what will lead to more therapeutic translation.25 Furthermore, for years, TM did not have any technical suggestions for improving translation, only two broad categories that were claimed to be essential for translatability: improving research infrastructure and broadening the goals of inquiry. This discrepancy has since been identified and efforts have been made to address it. For example, the EUSTM provided a textbook called Translational Medicine: Tools and Techniques as an initiative to provide concise knowledge to the fields stakeholders.24

Presently, TM has attracted considerable attention with substantial funding and numerous institutions and journals committed to its cause.25, 27 But before this, its arrival had to be incited. TM emerged in the late 1990s to offer hope in response to the shortcomings of evidencebased medicine and basic science research, such as the unsatisfactory results from the Human Genome Project, for instance.25 There were growing concerns that the explosion of biomedical research was not being translated in a meaningful manner proportionate with the expenditures and growing needs of the patients.27 The research had ignored what it took to properly disseminate new ideas.25 The difficulties of translation from bench to bedside have always been known, but what is different with TM is the amount of emphasis that is now put on translation and the recognition on how difficult and multifaceted it is to translate technologies.25 Over the past 20years, the role, power, and research volume of the field has increased, and TM is now a top priority for the scientific community.26 TM is also often used as common justification for research funding and conveys the message to politicians and taxpayers that research activities ultimately serve the public, which is also why it appeals to todays generation of students who want to work on big, realworld problems and make a meaningful difference.28, 29

As already mentioned, RM therapies are proving difficult to translate to the clinic.11 Although the basic research discoveries are never ceasing (books such as NewPerspectives in Regeneration by Drs. HeberKatz and Stocum30, and articles such as "Tissue Engineering and Regenerative Medicine: Past, Present, and Future" by Dr. Antnio Salgado et al.,31 provide comprehensive summaries of these advancements), therapy approval is practically nonexistent.30, 31, 32 This may be due, in part, to a tendency for people to blame the lack of translation of their technologies on extrinsic factors, thus removing responsibility.11 Additionally, the failures are not being studied. For example, stem cell research looks good in small animals but often fails in larger ones and then does not progress beyond phase II or III clinical trials because no benefits are found, and historically we have not been exploring why.11, 32 Consequently, the next therapies that are developed are improved by guesses rather than through a better understanding of the disease in mind (Figure).11

The negative feedback cycle currently present in most discovery and development processes of regenerative medicine. This cycle obstructs progression of the field.

RM has the potential to impact not only the quality of healthcare but also the economy, because the costs that could be avoided with curative therapies are immense.33 For this reason, analyzing the impact of RM on the translational strategy over time can help identify aspects that should be encouraged or discouraged to drastically improve translation. Reflecting on this history cannot only help us to avoid past mistakes but can also aid in redirecting the field to a onceproductive path.34 In the following section, the preclinical impact of RM on TM will be discussed, focusing on the shift from evidencebased medicine to trialanderror, the role of the basic scientist, and the emergence of the multidisciplinary approach. Clinical impact is also covered, concentrating on regulatory modifications. Last, changes in the private sector are considered as the shift in business models is detailed.

Because the RM field is essentially comprised of new ideas on cell renewal and tissue healing, it is logical that most of its impact would be on the preclinical side, as this is where ideas are tested, finetuned, and developed. Coincidentally, it is also where the translational strategy begins. Considering certain aspects early in the developmental process, such as realistic applications and ease of use, can help facilitate translation. RMs influence on TM can thus be separated into the three themes below.

Before the late 20th century, the majority of medical research was done using evidencebased medicine. This is a systematic approach to solving a clinical problem that integrates the best available research evidence together with clinical signs, patient values, and individual clinical experience all to support scientific decision making and research progression.35 As such, evidencebased medicine favors clinical trials and does not allow for much tinkering and only that which possesses highquality clinical evidence is to be pursued. This has its limitations, as it devalues mechanistic reasoning, and both in vitro and animal studies. Therefore, evidencebased medicine may have played a role in RMs downfall in the early 2000s. TE in the 1990s was using evidencebased medicine and was simply trying to copy tissue formation rather than trying to understand it.3 That most of the funding was coming from the private sector probably did not help either. Investors saw TE as an opportunity for quick returns on their investments, so therapies were rushed to clinical trials, which led to inconsistent results.14, 25, 32

As well, evidencebased medicine obscured the need for different methods of discovery. After RMs decline and the idea of TM came about, a trialanderror method was adopted. This technique favors a team effort, mechanistic reasoning, and seeks to change the social structure of research.25 Although clinical trials are still deemed important, the trialanderror method identifies that an idea needs to first be explored and should not necessarily require the confirmation of a hypothesis.11, 25 This new method is based more so on facts and has stimulated a more informed dialogue among stakeholders (whereas the confirmation or refusal of a hypothesis cannot always be made relevant to people outside the field). This, in turn, can help the regulatory agencies reduce the burden on their review boards in the evaluation and acceptance of novel strategies.11 Therefore, the failures of RM had helped to highlight the boundaries of evidencebased medicine and, combined with the rising intensity put on TM in the 1990s, assisted in defining the trialanderror based method.

Another thing that is changed with the historical development of RM has been the role of the basic scientist. Please see Figure for a summary of the differences between the traditional and modern scientist discussed in this review. Traditionally, basic scientists have worked with a discovery mindset, but without a noticeable regard for potential therapeutic applications. It has been noted that RM has made us realize how important it is to take the practical and industrializing aspects (like cost, for example) into account even at the basic research level.7, 14 The needs of the end users need to be considered during the developmental phase if RM is to establish a proper foothold within the market.15 In view of this, over the past 2decades, medical philosophy has changed in that it encourages basic scientists to communicate more with clinicians and vice versa. Experts like Barry Coller, MD, Vice President for Medical Affairs and PhysicianinChief at the Rockefeller University Medical Center, have identified various skills that a basic scientist must possess if translational research is to be improved.26, 28 Additionally, other researchers have commented that more and more basic scientists are motivated to have an impact on global health and this passion can be a source of inspiration that can help fuel interdisciplinary cooperation.28 Efforts have also been made to familiarize basic scientists with regulatory requirements. For example, the FDA publishes guide documents with recommendations on how to address these requirements.36 Despite this, much remains to be done, as there is still a lack of TM professionals and the current research environment hampers cooperation between experts (e.g., specialization is still encouraged, and achievement awards are individualized).26, 28

A comparison between the traditional and modern scientist. Although traditional scientists are more hypothesisdriven and rigid in terms of research methodology, if the concepts shown above are used, it can generate the modern scientist who is better suited for the translation of regenerative therapies. RM, regenerative medicine.

An additional point that can be argued is that because RM got basic scientists more involved in the translational process, this has consequently made them more realistic.37 As already mentioned, early RM therapies were comprised of complex cell therapies that were not fully understood. From 2004 onward, the field diversified to include research into simpler acellular products.38 Other avenues, such as induced pluripotent stem cells, endogenous repair, nanotechnology, and regenerative pharmacology, are also being explored.37, 39, 40, 41 Increasingly, experts are trying to spread this message; for instance, in the field of cardiology, Dr. Mark Sussman, a world renowned cardiac researcher, and his colleague Dr. Kathleen Broughton at the San Diego State University Heart Institute and the Integrated Regenerative Research Institute, recently stated that After over a decade of myocardial regenerative research studies, the initial optimism and enthusiasm that fueled rapid and widespread adoption of cellular therapies for heart failure has given way to more pragmatic, realistic, and achievable goals.9

The last preclinical impact of RM to be discussed is the arrival of the multidisciplinary approach. This now widespread notion identifies that to improve translation and accelerate technology development, it is better to have a team composed of experts from multiple disciplines, because the various backgrounds and schools of thought can be combined with each contributing to a project in a different way.25, 39 What has surely incited its evolution is that RM inherently requires contributions from biologists, chemists, engineers, and medical professionals. This need has led to the formation of institutions that house all the required expertise under the same roof (such centers have increased in number since 2003), which promotes more teamwork between laboratories and clinics.28 Dr. Jennifer Hobin et al.28 states that bringing dissimilar research expertise together in close proximity is the key to creating an environment that facilitates collaboration. In addition, it could be said that these collaborative environments help minimize the flaws of medical specialization, which occurred in the second half of the 19th century; where the ideological basis that the human body can be categorized combined with the rapid arrival of new medical technologies led to the specialization of medical practice, which, in turn, led to the segregation of medical professionals from each other and the patient.42 Coincidentally, if one recalls the definition of TM, it, along with the trialanderror based method, suggests that improved research infrastructures and team efforts can facilitate the translation of therapies.

We now look at the influence that RM has had on the clinical side of therapy development. Before the subject is discussed, it is important to note that the reason clinical research has been affected is because of the uniqueness of RM therapies. Their novelty does not fit within the current regulatory process or use in clinical trials, and although the latter has yet to adapt, the regulatory sector has attempted over the years to facilitate the journey from bench to bedside.7, 43, 44

Initially, when RM was in its infancy, its therapies were regulated by the criteria originally developed for drugs; and as we have seen, this was identified as a factor that led to its downfall. Now, in 2019, several regulatory changes have been implemented to rectify this. What has helped has been the input from other countries. As mentioned above, RM started in the United States, but after the crash, other countries like the United Kingdom and Japan caught up, and their less stringent regulatory procedures have allowed them to better adapt the framework for these new therapies.22 In 2007, the European Union passed the Advanced Therapy Products Regulation law, which defined regenerative therapies, categorized them, and provided them with separate regulatory criteria for advanced approval.13, 43 In 2014, public pressure and researcher demands led Japan to enact three new laws: the Regenerative Medicine Promotion Act, the Pharmaceuticals, Medical Devices, and Other Therapeutic Products Act, and the Act on the Safety of Regenerative Medicine. These unprecedented national policies now help therapies gain accelerated and conditional approval to better conduct clinical trials and to better meet the demands of the patients.7, 13, 44, 45 During this time, the United States has not stood idle. In 2012, the US Congress passed the FDA Safety and Innovation Act (FDASIA), which expanded its existing Accelerated Approval Pathway to include breakthrough therapies, a category created for new emerging technologies, including regenerative strategies.13, 46 Drs. Celia Witten, Richard McFarland, and Stephanie Simek provide a wellwritten overview on the efforts of the FDA to accommodate RM.36 By and large, it is safe to say that RM has spurred a drastic change in traditional regulatory pathways to not only better manage these novel therapies but also put more weight on efficient translation.

It is also important to discuss changes in the private sector because manufacturing and marketing is and will remain one of the greatest obstacles facing RM, and, once again, the novelty of the field is responsible. Although the bulk of the problems remain, there has nonetheless been a change in business strategies that is worth appreciating.

Throughout its history, RM research has been carried out by academic research institutions or small and mediumsized enterprises.23, 47 With this in mind, the business model used in the health industry varies depending on the type of company. The royalty model is the one primarily used by biotech companies.8, 14 Here, businesses will develop a therapy up to the clinical stage and then hand it off to a company with more resources (usually a pharmaceutical one) who can carry out the larger scale studies. With this model, biotech companies make money simply through royalties and this carries both pros and cons (Figure).

A comparison of both the royalty and integrated business models used by private companies in the biomedical industry. The pros and cons are listed with the assumption that they are for a startup company in regenerative medicine.

Because the market for regenerative therapies currently is not big enough for the royalty model, startups have had to shift to an integrated model where the discovery, development, approval, and manufacturing of a new therapy are all done internally (which is unusual for small startups).8 Using this strategy, the companies can reap all the rewards but obviously also assume all the risk.

The market for regenerative therapies has so far been small enough that smaller firms do not have to manufacture large quantities of their products (like they do in the pharmaceutical industry) and they can start making money in a quicker fashion.8 Whether the business model will change again as the market grows or if the original startups will grow in proportion remains to be seen.14 What is to be highlighted here is that those who seek to commercialize regenerative therapies have had to shift to an integrated business model (that was not previously the norm for smaller ventures), which has affected translation by letting them have more influence in determining how their therapy is being developed, marketed, and manufactured.

Having detailed RMs relationship with the translation strategy and the aspects that changed in conjunction with the fields development, the remainder of the review will summarize the challenges that are contributing to RMs modest performance in the clinic.

With increased funding and a growing number of committed institutions, many countries have become increasingly invested in RMs success. For example, the US Department of Health and Human Services recognizes RM as being at the forefront of healthcare.7 As well, the UK government has identified RM as a field in which they can become global leaders and that will generate significant economic returns.44 The literature indicates that RM is reaching a critical mass and is on the verge of a significant clinical transition. The optimism is as high as it has ever been and the rush to succeed with clinical trials is equally felt.23 However, the bottom line is that the clinical and market performance is still very poor. Being that a gold standard for treatment in RM remains elusive, clinicians are often illinformed about current applications, and studies on safety and efficacy are lacking.23, 44, 48, 49 The National Institute of Health estimates that 8090% of potential therapies run into problems during the preclinical phase.28 Naturally, scientists have offered various explanations for these results, such as deficiencies in translational science and poor research practices in the clinical sciences.50 Shockingly, in a 2004 analysis, 101 articles by basic scientists were found that clearly promised a product with major clinical application, and yet 20years later, only 5 were licensed and only 1 had a major impact.50 Therefore, it is easily deducible that many challenges still lie ahead. The perceived riskbenefit ratio remains high and, as a consequence, clinical trials have been proceeding with caution.13, 23, 33 Numerous reviews have been published on these challenges but with an emphasis on those relating to the clinical phase.11, 13, 22, 51 Although these will be summarized below, the present study highlights the identification and analysis of the preclinical challenges. Please see Figure for a summary of the preclinical and clinical obstacles discussed herein.

Summary of the preclinical and postclinical challenges discussed. Even though preclinical obstacles to the translation of regenerative medicine therapies are more elusive, they are just as significant as their counterparts.

To begin, a possible explanation for the preclinical obstacles being underrepresented in the literature is because of the pliability of the phase itself. Although the clinical phase is composed of numerous subphases and strict protocols, the preclinical research is much less structured with less oversight. Whereas rigorous scientific method is applied to the experiments themselves, which usually consist of in vitro followed by in vivo experiments, the basic scientist has more flexibility regarding experimental organization, structure, and backtracking; thus, making explicit challenges possibly harder to recognize.

Some researchers have nevertheless attempted to do so. For example, Dr. Jennifer Hobin et al. have identified three major risks associated with RM technologies as being tumorigenicity, immunogenicity, and risks involved with the implantation procedure.13 The first two relate to arguably the largest preclinical challenge, which have been identified as needing a better understanding of the mechanism of action.12 Although the difficulties of identifying a mechanism are appreciated in the scientific community, it is imperative that improvements in this area are made as it will affect application and manufacturing decisions. Hence, greater emphasis on identifying the mechanism of action(s) will need to be adopted by basic scientists who are looking to develop a technology.

Another significant preclinical challenge is the lack of translation streamlining for basic scientists. Although basic scientists have become more involved in the translational process and more pragmatic over the years, there is, in general, still a lack of incentive and available resources to help a scientist translate their research. Academic faculty members are given tenure and promotion based on funding success (grants) and intellectual contributions (publications).28 Thus, researchers who have received money to conduct research and publish their work on a promising new therapy might stop short of translation as there may be no additional recognizable accomplishment or motivation for such an endeavor. For example, Jennifer Hobin et al. described the case of Dr. Daria MochlyRosen at Stanford Universitys Translational Research Program, who sought help for an interesting idea for a heart rate regulation therapy.13 She was turned down by numerous companies that found the clinical challenges too daunting and her colleagues offered no support but rather discouraged her from pursuing the idea saying that it would not be worthwhile for her career.

Last, a very important preclinical challenge that has gained recognition over the past few years is the lack of appropriate preclinical testing models. It is often reported that novel therapies that do well in the laboratory but then fail in larger animal studies or clinical trials. This is partly due to a lack of mechanistic insight, but also because of a shortage of appropriate in vitro, in vivo, and ex vivo models.9, 36 With properly validated preclinical models, we would be better able to gauge the performance of novel therapies and predict their future clinical success, but instead we are misidentifying the potential of therapies. Notably, the lack of appropriate models also contributes to the difficulty in obtaining reliable data on the underlying mechanism(s) of action of RM therapies, as differences may exist between the preclinical and clinical settings.

As far as clinical challenges go, they are numerous. Stem cell trials in particular have received criticism from a perceived lack of rigor and controlled trials.23 Related to this, a potent point that has arisen over the past few years is the absence of longterm followup studies for clinical trials, which is clearly necessary to establish the safety and efficacy of these interventions.13, 33 Unfortunately, they are costly and they are timeconsuming. Efforts are nonetheless being made to overcome these obstacles. For example, in 2015, the Mayo Clinic released an RM buildout perspective offering a blueprint for the discovery, translation and application of regenerative medicine therapies for accelerated adoption into standard of care.7 Institutions, such as Canadas Center for Commercialization of Regenerative Medicine, have been launched to help researchers mitigate the risks of cell therapy development by offering technical as well as business services.12, 51 Experts are also stepping up; for example, Drs. Arnold Caplan and Michael West proposed a new regulatory pathway that incorporates large postmarket studies into clinical trials.33

In terms of manufacturing, it is difficult to engage industry because the necessary technology to produce RM therapies at an industrial level does not exist yet. Scaleout and automated production methods for the manufacturing of regenerative therapies are needed.7, 10, 12, 23, 52, 53 This challenge stems from the complexity and natural intrinsic variation of the biological components, which makes longterm stability difficult to achieve and increases manufacturing costs.13, 44 Now, if RM therapies could establish their superiority over conventional treatments, then this would potentially alleviate costs and increase the likelihood of being reimbursed, but it remains to be seen.13 A hot topic at the moment is the choice between autologous or allogeneicbased products, which would entail either a centralized or decentralized manufacturing model, respectively (although hybrid models have been proposed).7, 23, 54 Autologous products, being patientspecific, have the advantage of having smaller startup costs, simpler regulations, and point of care processing.47 As for allogeneic products, they are more suitable for an off the shelf product, for a scaleout model and quality controls can be applied in bulk.47, 54 Dr. Yves Bayon et al.51 provided a thorough description of this topic while simultaneously indicating areas that have been identified for improvement.

As mentioned above, regulatory challenges are what have been most addressed thus far through scientific and public pressure. Moving forward, the goal identified by expert thinktank sessions is to harmonize RMspecific regulations across agencies and countries.7, 36 Reimbursement is the last of the regulatory challenges to be considered. In order for RM treatments to become broadly available, reimbursement is a necessity and both public and private healthcare need to determine how the regulations will be modified for disruptive therapies coming down the pipeline.13, 23, 44

RM has had an undeniable influence on the process of bench to bedside research. Preclinically, it has helped identify the limitations of evidencebased medicine and contributed to the paradigm shift to the trialanderror method. Likewise, the field has changed its mindset and the basic scientist is adopting new responsibilities becoming more motivated, pragmatic, and involved in TM, rivaling researchers in the applied sciences. The multidisciplinary approach has also been promoted by RM over the years and institutions dedicated to fostering collaborative research in RM have increased in numbers. Clinically, regulatory pathways that were developed for drugs and biomedical devices, and which have been in place for decades, have been adapted to aid RMs disruptive technologies, leading to new guidelines that favor translation. In the private sector, the novel nature of RM therapies has led to startup companies using an alternative business model that provides them toptobottom authority over the development of their products and it is yet to be seen if the business strategy in place will be sufficient as the industry grows.

If the translation of RM therapies is to be improved, many of the challenges to be overcome lie in the early stages of therapy development, such as identifying the mechanism(s) of action, validating preclinical experimental models, and incentivizing translational research for basic scientists. In later stages, regulatory changes have been made, but much still needs to be addressed. This includes the adoption of clinical trials that are more rigorous and include longterm followup studies, the development of appropriate manufacturing technology, the synchronization of regulatory agencies, and a clear plan for reimbursement strategies. Once again, these challenges have been discussed in greater detail in previous works.2, 3, 7, 12, 13, 15, 22, 23, 26, 31, 38, 44, 48, 51, 52 While it seems that the field may be at a tipping point with many challenges remaining, the fact that translation has been influenced in a positive way gives promise to the future progression of RM therapies.

This work was supported by a Collaborative Research Grant from the Canadian Institutes of Health Research (CIHR) and the Natural Sciences and Engineering Research Council (NSERC; CPG158280 to E.J.S.), and the Hetenyi Memorial Studentship from the University of Ottawa (to E.J.).

All authors declared no competing interest for this work.

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