FINDINGS
Scientists from UCLA and the Howard Hughes Medical Institute successfully used a gene that suppresses cholesterol levels as part of a treatment to reduce plaque in mice with a disorder called familial hypercholesterolemia. In a preclinical study, researchers found that the gene, LeXis, lowered cholesterol and blockages in the arteries, and the treatment appeared to reduce the build-up of fat in liver cells.
Familial hypercholesterolemia is an inherited condition characterized by extremely high levels of low-density lipoprotein cholesterol (commonly referred to as bad cholesterol) and an increased risk of early heart disease.
The LeXis gene belongs to a unique group of genes that until recently were considered junk DNA because scientists believed they served little purpose. However, evidence from the human genome project led to the discovery that genes like LeXis are actually active. The study of these genes,now referred to as long noncoding ribonucleic acids, or lncRNAs, is a rapidly evolving area in biology.
Researchers wanted to test whether a single injection of LeXis could slow the development of heart disease. To do so, they gave the mice either LeXis or a control gene, and fed them a 15-week diet consisting of food high in sodium and cholesterol the mouse equivalent of fast-food hamburgers and french fries. Researchers then measured the progression of heart disease.
In the next phase of the study, researchers intend to confirm the findings in larger animals and test the therapy in combination with currently available treatments.
Although previous research has shown that lncRNAs can be important, this is the first study to show that they could potentially be used to treat a human disease using gene therapy. Junk genes could one day offer a framework for treating people with familial hypercholesterolemia and other conditions that are otherwise very difficult to treat.
The papers authors are Xiaohui Wu, Zhengyi Zhang and Dr. Tamer Sallam of UCLA; and Dr. Peter Tontonoz, Marius Jones and David Salisbury of the Howard Hughes Medical Institute.
The study waspublished onlinein the journal Circulation.
The research was supported by grants from the National Heart, Lung, and Blood Institute; the American College of Cardiology; and the Lauren B. Leichtman and Arthur E. Levine UCLA Cardiovascular Discovery Fund.
Learn more about the cardiovascular research theme at UCLA.
More here:
Gene therapy using 'junk DNA' could lower risk for heart disease - UCLA Newsroom
- Krystal Biotech Inc (KRYS) Reports Strong Q4 and Full Year 2023 Financial Results - Yahoo Finance - February 27th, 2024
- New sickle cell gene therapies are a breakthrough, but solving how to pay their high prices is a struggle - CNBC - February 27th, 2024
- 10 gene therapy companies you should know about - Labiotech.eu - February 27th, 2024
- Former Penn employee sues Gene Therapy Program director, alleging 'unethical and illegal conduct' - The Daily Pennsylvanian - February 27th, 2024
- Gene therapy can't cure deafness because it doesn't need to be cured - The Philadelphia Inquirer - February 27th, 2024
- Fetal surgery expert Tippi MacKenzie shifts to in-utero gene therapy - STAT - February 27th, 2024
- Therapeutic Solutions International Files Patent on Facilitating Effects of JadiCells on Gene Therapy Mediated Cell ... - Business Wire - February 27th, 2024
- CU Anschutz Set to Launch Gene Therapy Trials for Heart Disorder - University of Colorado Anschutz Medical Campus - February 27th, 2024
- ICH targets cell and gene therapies, real-world data as topics ripe for harmonization - Regulatory Focus - February 27th, 2024
- Encoded Therapeutics Announces UK CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101 - Business Wire - February 27th, 2024
- Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich's Ataxia in ... - GlobeNewswire - February 27th, 2024
- Therapeutic Solutions International Files Patent on Facilitating Effects of JadiCells on Gene Therapy Mediated Cell ... - The Bakersfield Californian - February 27th, 2024
- Ring Therapeutics, a Flagship gene therapy startup, lays off 20% of staff - STAT - February 27th, 2024
- Ed Silverman on new gene therapies, biosimilars, coffee, and more - STAT - STAT - February 27th, 2024
- Combatting HIV using gene therapy - Drug Target Review - February 27th, 2024
- NCDHHS working with federal government on increasing access to sickle cell disease treatment - WECT - February 27th, 2024
- Cell Therapy Weekly: FDA Approves First Cell Therapy For Solid Tumors - RegMedNet - February 27th, 2024
- Rejuvenate Bio Announces Gene Therapy-Mediated Partial Reprogramming Extends Lifespan and Reverses Age ... - Business Wire - February 27th, 2024
- New sickle cell therapy uses gene editing at MUSC | Health | postandcourier.com - The Post and Courier - February 27th, 2024
- Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency - Nature.com - February 18th, 2024
- Gene therapy, blood transfusions, stool monitoring: The Sydneysiders trying to escape death - Sydney Morning Herald - February 18th, 2024
- Not too late to repair: gene therapy improves advanced heart failure in animal model - Baylor College of Medicine | BCM - February 18th, 2024
- Direct Administration of CRISPR-Cas9: Tools for Cell and Gene Therapy - Forbes - February 18th, 2024
- Gene therapy's promise seen in its success for AADC deficiency - AADC News - February 18th, 2024
- First Patient Randomized in AskBio Phase II Gene Therapy Trial for Congestive Heart Failure Treatment - Diagnostic and Interventional Cardiology - February 18th, 2024
- FDA to review expanded use of Sarepta Duchenne gene therapy - Yahoo Finance - February 18th, 2024
- How gene therapy can help treat a rare blood disorder - KUTV 2News - February 18th, 2024
- CRISPR gene therapy Casgevy secures approval in Europe - Pharmaceutical Technology - February 18th, 2024
- FDA Updates for the Week of Feb. 12, 2024: New Approvals, an Extended Review and Goal Dates Set - Managed Healthcare Executive - February 18th, 2024
- First Patient Randomized in AskBio Phase II Gene Therapy Trial for Congestive Heart Failure - Business Wire - February 18th, 2024
- Lisa Jarvis: A cure for some kinds of hearing loss? Gene therapies are getting closer - St. Paul Pioneer Press - February 18th, 2024
- Spinal Muscular Atrophy Market: Navigating Therapeutic Advances towards USD 5.6 Billion by 2034, with a CAGR of ... - Yahoo Finance - February 18th, 2024
- Crispr Therapeutics' Gene Therapy Wins EU Approval - TipRanks.com - TipRanks - February 18th, 2024
- Revolutionizing Gene Therapy: A Novel Technique for In Vivo CAR T Cells - Medriva - February 18th, 2024
- How Stella Vnook is 'infusing innovation' into cell therapies with Likarda's technology - BioPharma-Reporter.com - February 18th, 2024
- Using cancer's 'superpower' to boost T-cell therapies in mice - FierceBiotech - February 18th, 2024
- Parkinson's Disease Patient Identification and Education Will Need to Improve When New Disease Modifying ... - Yahoo Finance - February 18th, 2024
- CRISPR (CRSP) to Report Q4 Earnings: What's in the Cards? - Yahoo Finance - February 18th, 2024
- Gene Therapy: A Ray of Hope for Heart Failure Treatment | Health News - Medriva - February 18th, 2024
- Gene therapy trials restore hearing to children born deaf - The Hill - February 2nd, 2024
- Early Trial Success for Inherited Deafness Gene-Therapy - RegMedNet - February 2nd, 2024
- Sickle cell gene therapy first focus of Medicaid pay model - Modern Healthcare - February 2nd, 2024
- Beyond chemotherapy: Navigating the promise and perils of CAR-T therapy in pediatric cancer - BioPharma-Reporter.com - February 2nd, 2024
- Angiodema: Gene therapy blocks painful hereditary disorder - BBC - February 2nd, 2024
- FDA: Genome editing therapies may use accelerated approval pathway - Regulatory Focus - February 2nd, 2024
- CRISPR tech treats severe hereditary condition successfully - Interesting Engineering - February 2nd, 2024
- Gene Therapy Restores Hearing in Small Trial for Kids With Hereditary Deafness - Technology Networks - February 2nd, 2024
- Gene-based therapy may slow development of life-threatening heart condition - Medical Xpress - February 2nd, 2024
- Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum ... - Business Wire - February 2nd, 2024
- Replacing the PKP2 gene prolongs survival in ARVC patients - Drug Target Review - February 2nd, 2024
- Cell and gene therapies top pharmaceutical industry trend to watch for 2024 - Pharmaceutical Technology - February 2nd, 2024
- Gene Therapy Restores Hearing in Children with Hereditary Deafness - The Hearing Review - February 2nd, 2024
- 2 Dutch among 10 "functionally cured" of hereditary swelling disease by new gene therapy - NL Times - February 2nd, 2024
- FDA publishes final industry guidance for CAR-T and gene therapies - PMLiVE - February 2nd, 2024
- Gene therapy cures hereditary deafness in two people - Advanced Science News - February 2nd, 2024
- Hearing Restored in Deaf Children in Gene Therapy Clinical Trial - Securities.io - February 2nd, 2024
- Vivet Therapeutics Receive 4.9 Million to Advance Development of Gene Therapy - European Pharmaceutical Manufacturer - February 2nd, 2024
- Adverum Biotechnologies will Host Webcast to Review LUNA Phase 2 Preliminary Efficacy and Safety Results on ... - GlobeNewswire - February 2nd, 2024
- Allogene parks Notch CARs, leaving one on the road - FierceBiotech - February 2nd, 2024
- Experimental gene therapy allows kids with inherited deafness to hear - The Associated Press - January 25th, 2024
- Gene-therapy breakthrough allows congenitally deaf children to hear Harvard Gazette - Harvard Gazette - January 25th, 2024
- Gene therapies restore hearing in several kids with inherited deafness - Livescience.com - January 25th, 2024
- Boy born deaf can hear for the first time thanks to a gene therapy procedure - NBC 6 South Florida - January 25th, 2024
- Gene therapy restores hearing in children with hereditary deafness - Medical Xpress - January 25th, 2024
- Children's Hospital of Philadelphia Performs First in U.S. Gene Therapy Procedure to Treat Genetic Hearing Loss - PR Newswire - January 25th, 2024
- Children's Hospital of Philadelphia uses gene therapy to help 11-year-old boy hear for 1st time - WPVI-TV - January 25th, 2024
- Breakthrough gene therapy treatment restores hearing in children born deaf - The Independent - January 25th, 2024
- Gene Therapy Administered Via Virus Cures Deafness in 11-Year-Old Boy - Futurism - January 25th, 2024
- 11-Year-Old Boy Hears For First Time Ever Thanks To Gene Therapy Breakthrough - IFLScience - January 25th, 2024
- Gene Therapy Deafness | National | caledonianrecord.com - The Caledonian-Record - January 25th, 2024
- Sickle cell gene therapy to be covered by Blue Cross insurers - Modern Healthcare - January 25th, 2024
- Experimental gene therapy allows kids with inherited deafness to hear - Jackson Hole News&Guide - January 25th, 2024
- Gene therapy allows 11-year-old boy to hear for the first time - Latest From ITV News - ITVX - January 25th, 2024
- Experimental Gene Therapy Allows Kids With Inherited Deafness To Hear - Southern Minnesota News - January 25th, 2024
- Joint US-China gene therapy trials restore hearing in 5 kids, a first - Interesting Engineering - January 25th, 2024
- Gene Therapy Deafness | Nation | The Daily News - Galveston County Daily News - January 25th, 2024
- In Draft Guidance for Cell and Gene Therapy Industry, FDA Broadens its Approach to Product Potency - Precision Medicine Online - January 25th, 2024
- Gene therapy helps restore hearing in children with hereditary deafness - Shropshire Star - January 25th, 2024
- Gene therapy breakthrough enables deaf boy to hear for the first time - The Week - January 25th, 2024
- FDA eyes collaborative review pilot for gene therapies - Regulatory Focus - January 17th, 2024