StemCell Therapy

Cancer treatments are improving as scientists are finding ways to develop new techniques and treatments. One of which is precision medicine, where they have focused on improving patients health using personalized solutions.

RELATED: Oxfords Genomics Pushing the Boundaries of Personalized Medicine

Precision medicine, in the simplest definition, is the way a patient is treated, diagnosed, or prevent disease by checking his/her genetics, environment, or lifestyle.

This type of treatment is related to pharmacogenomics. Where pharmacogenomics is the study of how a persons gene affects his/her response to a drug, it is used to treat a person through effective and safe medication tailored to their genes.

Precision medicine is now commonly used on patients treated with pancreatic cancer, lung cancer, melanoma/skin cancer, and colon cancer. It is also used to detect and treat HIV and cystic fibrosis.

Slowly, it is also seen in treatments for heart diseases, Alzheimers disease, rheumatoid arthritis, and multiple sclerosis.

In cancer patients, most medical facilities treat every patient the same way. However, studies suggest that not everyone responds to treatments the same way. One persons body may react differently with medicines as compared to another person.

Genetics plays a role in treating tumors, and precision medicine promise to tailor treatments based on a persons genes. It is seeing how a tumor would react to certain treatments that may work for other people.

Precision medicine can be used in the prevention and prediction of disease and management and treatment. Here are some examples of how it is used to treat, prevent, or treat people in a practical setting.

Checking your familys history of diseases and illnesses can somehow determine what you are capable of acquiring. If a family member has a history of cancer, heart diseases, diabetes, high blood pressure, or other chronic diseases, there is a high chance of you getting it.

With this data and information, a doctor can create treatment plans to prevent these from happening to you.

For example, when the doctor finds out that any of your family members had breast cancer, then the chances of you having it is likely. The doctor will then decide for you to have regular mammograms to check for any signs.

Newborns (usually right after theyre conceived) are screened where blood samples are taken. This test will check if they have any pre-existing conditions acquired from their parents, check hearing capabilities or heart defects, among others.

This way, the baby will be treated accordingly if any crucial or life-threatening conditions are seen.

For example, the newborn screening shows Baby Mary has severe combined immunodeficiency (SCID), she will receive a bone marrow transplant immediately to battle her condition. SCID is life-threatening to babies since its responsible for fighting off infections.

Personal trackers such as smartwatches or other mobile devices that check on your health can be lifesavers and be tools for precision medicine.

For example, a person is notified by his smart device that he is experiencing abnormal heart rates even if he has no family history of any heart condition. He then goes to see a doctor because of this and has been diagnosed with atrial fibrillation. This device could have saved his life because that condition can lead to a stroke. Now, he can treat his condition before it worsens.

Genomic sequencing can be used to control and track-out infectious diseases. Similar to whats been used to track COVID-19, this approach shows a DNA of a germ or virus where scientists have the opportunity to learn more about it and find a treatment a cure for it.

An example of this is the COVID-19, where scientists were able to extract samples from those infected with the virus and learn about it and find vaccines and cures for it, which is now slowly happening to us.

As a treatment, tumor profiling is genetic testing of a tumor. It is a way for doctors to choose which kind of treatment they would use for a condition. They would know from this process if cancer will return or would need radiation or chemotherapy.

For example, Jennys breast cancer returned and is diagnosed again. But her tumor profiling reveals she has triple-negative breast cancer. Her approach to this, along with her doctors, is a more aggressive one, including chemotherapy, radiation, and mastectomy.

RELATED: FDA Approves GSKs Checkpoint Inhibitor Jemperli for Endometrial Cancer

As mentioned above, pharmacogenomics studies how a person reacts to a certain treatment based on their genes. Doctors using this treatment can gauge if a certain medicine can be effective or not based on a patients history. They can also determine if the patient will experience any serious side effects.

For example, John needs to undergo Fluorouracil (5-FU), which is a type of chemotherapy. But if John has a low level of an enzyme called dihydropyrimidine dehydrogenase (DPD), which helps metabolize fluorouracil in the body, the doctors would need to check on him using pharmacogenomics. If he has a low dose of fluorouracil, an oncologist will decrease the dosage in the chemotherapy to prevent any serious side effects.

With these examples revealed, some facilities and companies provide precision medicine to improve the living conditions of patients treated with different diseases.

ExactCure is a French start-up that combines artificial intelligence with precision medicine to create flawless software for the use of drugs to be used by patients depending on their kidney status, genotype, gender, or age.

Patients use this service by inputting their data, and ExactCure will give the necessary medications based on the information provided.

Tepthera is a Swiss start-up that focuses on cancer immunotherapy, infectious and auto-immune diseases.

Their focus concerning precision medicine is on identifying T cell antigens for better and personalized therapies and treatment.

Caris Life Sciences is a molecular science company that focuses on precision medicine in oncology. They are working on the development of innovative therapeutics and advance potential treatments for cancer in the clinic.

They develop profiling assays for oncology that scan DNA, RNA and proteins to reveal a molecular blueprint to help physicians determine the best course of treatment for cancer patients.

Precigen is a Maryland-based company that is advancing its UltraCAR-T cell therapy approach to treating cancer.

They are now developing next-generation gene and cell therapies that can change the treatment paradigm in immuno-oncology, autoimmune disorders and infectious diseases.

There are numerous ways to treat diseases and medical conditions with the use of precision medicine. Scientists are continually finding out ways to improve patients lives by using their traits.

Read more:
Precision Medicine: Improving Health With Personalized Solutions - BioSpace

Across metro Portland, 22 students from 10 public and private high schools have won the coveted $2,500 National Merit scholarships given to students who show exceptional promise.

Half of those 2021 winners attended just two high schools: Sunset and Westview, both in the Beaverton School District.

Three other high schools had two winners each: the private Jesuit and Catlin Gabel schools and Lake Oswego High.

Winners of the $2,500 scholarships are judged the best of the best among National Merit Scholars, with what the scholarship organization says are the strongest combination of accomplishments, skills and potential for success in college. Panels of high school counselors and college admission officers pick them based on grades, difficulty of courses taken, standardized test scores, an essay and recommendations from a teacher and their high school.

Normally, as happened this year, National Merit officials limit that to 26 students across Oregon. In addition to the 22 winners from the Portland area, students in Ashland, Bend, Eugene and Silverton also made the elite achiever list.

The National Merit Corporation provides extremely limited information about winners, and The Oregonian/OregonLive found most of this information through independent research. Some school officials, including those at Sunset High, did not respond to requests for information about their winning students.

Here are the winning Portland-area students with additional information as available:

Catlin Gabel

Sophie Kruse, senior class president at Catlin Gabel, is headed to Columbia University to study engineering.

Sophia Kruse

She was a state leader in the Students Demand Action office, which mobilizes and educates voters about gun violence, and was elected senior class president at Catlin Gabel. She competed for four years as a member of Lincoln Highs varsity lacrosse team and was named player of the year in Portland Public Schools her senior year. She competed on Catlins cross country team and its soccer team, which won two state championships. She worked on her schools environmental action team all four years of high school. She will attend Columbia University, where she plans to study engineering.

Tiffany Toh, a standout in debat and robotics, is headed to Yale University.

Tiffany Toh

During high school, she was interested in music and debate and was known for being extremely energetic, according to the Catlin website. She served as a three-year member and eventual co-leader of the winning robotics team, The Flaming Chickens. She was a member of the varsity swim team and served as a school ambassador. As a senior, she was named one of 10 Deans List students among 90,000 who competed in events leading to the international high school robotics competition. She is headed to Yale University and told National Merit officials she is likely to major in biomedical engineering.

Krishna Home school

Gopal Goel, who placed fourth in the nation's oldest and most prestigious math and science competition for high school seniors, is headed to MIT to study math and physics.

Gopal Goel

He won $100,000 the nations oldest and most prestigious science and math competition for high school seniors for math research that made connections between two subjects regarding randomness and probability. He told contest organizers he hopes his work will aid in the search for the true nature of quantum gravity, more commonly known as the theory of everything. He can solve a Rubiks cube even while blindfolded. He won a silver medal at the 2020 International Math Olympiad. He mentored students on the U.S. Physics Olympiad team. He is headed to the Massachusetts Institute of Technology, better known as MIT, and plans to study math and physics.

Jesuit

Eliza Daigle, who reveled in lacrosse, science and volunteerism during high school, is headed to Whitman College, where she plans to study biochemistry and related fields.

Eliza Daigle

She earned class academic achievement awards in science and Spanish. She served as co-captain of the womens varsity lacrosse team, which won its first state championship in 2019 and won again this year. She took part in Oregon Health & Science Universitys partnership in scientific inquiry program, which introduces high school students to the scientific process and laboratory research. In turn, she served as a co-leader of Jesuits junior scientists program, which aims to encourage a love of science through fun experiments that develop problem solving and critical thinking skills. She volunteered at Trinity Episcopal Cathedral and contributed more than 100 hours of service to Good Samaritan Medical Center. She is headed to Whitman College, where she will play lacrosse and plans to major in biochemistry, biophysics and molecular biology.

Passionate about computer science, problem solving and research, Marissa Kuo is headed to Santa Clara University to study computer science and engineering.

Marissa Kuo

She received the outstanding applied chemistry award from the Portland chapter of the American Chemical Society at the Northwest Science Fair for her research on green chemistry methods for degrading neurotoxic herbicides. She interned at Portland State University in a computer science lab where she studied computer algorithms for night vision. She helped lead her schools Asian Student Union and research club. She competed on the Jesuit swim team. Passionate about computer science, problem solving, and research, she is headed to Santa Clara University to study computer science and engineering.

Lakeridge

Roselyn Dai

She volunteered as a peer intervention specialist at the Portland-based teen crisis hotline YouthLine and was honored by the Oregon Chinese Coalition for her volunteerism. She also volunteered at two local medical centers and was a multi-year member of her schools speech and debate team. She told National Merit officials she is likely to study medicine.

Lake Oswego

Jackie Booth, who played varsity tennis and taught younger students math for three years, is headed to the University of Southern California.

Jaqueline Jackie Booth

She competed all four years on the varsity tennis team and volunteered regularly at Portland Rescue Mission and other community organizations. She co-led the schools fellowship club and worked as a math instructor at Mathnasium Learning Center since her sophomore year. She is headed to the University of Southern California, where she plans to major in philosophy, politics and law.

George Danzelaud, a poet, violin player, singer-songwriter and varsity sprinter, is headed to the University of Southern California to study business and computer science.Randal Leitch Photography

George Danzelaud

A varsity sprinter who loves both science and the arts, he was elected president of his senior class. He performed a virtual concert with his brother to raise money for the Oregon Humane Society in the wake of last summers deadly wildfires. He performed solo violin at Carnegie Hall, won a gold award for his poetry and is a self-taught guitarist and singer who published three songs on iTunes and Spotify. He is headed to the University of Southern California and plans to double major in computer science and business.

Oregon Episcopal School

Lila Schweinfurth

She was the state winner of U.S. Stockholm Junior Water Prize. Her research on predicting harmful algal blooms to improve water quality also put her among the five Oregon students named semifinalists in the nations oldest and most prestigious math and science compeitions for high school seniors. She plays violin and plans to study science and prepare for a career involving research, according to National Merit officials.

Sunset

Anika Gupta is passionate about the real-world applications of science and technology and has deeply researched aquaponics in pursuit of sustainably growing food. She will attend the University of Southern California, where she plans to double major in business and computer science.

Anika Gupta

Anika Gupta is passionate about equity and real-world applicability of science and technology. Four years ago, she started a classroom aquaponics project that shes since grown into a bioengineering syllabus used by more than 700 student researchers. Shes working alongside the NASA Ames Research Center to learn to grow food sustainably. Anika also co-founded Project Portland Carry, a nonprofit to support students during COVID through educational helplines and accessible lunch delivery. As president of the Sunset speech and debate club, Anika rebuilt her club amid what she called existential challenges and led Sunset to a state championship victory this spring. She personally was named Oregons top extemporaneous speaker. She is also a recipient of the prestigious Coca-Cola Scholarship. She is headed to the University of Southern California, where she plans to study business and computer science.

Amuthan Amu Ilavarasan

He won a scholarship for winning an art competition. He designs and sells t-shirts. He told National Merit officials he was likely to study environmental science.

Eric Kim conducted two years of research on bioinformatics at Oregon Health & Science University, plays clarinet and is a competitive Taekwondo sparring athlete. He is headed to Columbia University.

Eric Kim

He founded and led the Sunset Science Ambassadors program that matches high school science scholars with fifth-graders to mentor them through hands-on science projects. He is one of five Oregon students named a Regeneron Scholar for his biology research as part of the nations oldest and most prestigious science and math competitions for high school seniors. A longtime volunteer for hard-of-hearing individuals, he designed see-through face masks to aid their communication during the pandemic and helped people all over the country get materials and instructions needed to make them. He conducted two years of research on bioinformatics at Oregon Health & Science University, plays clarinet and is a competitive Taekwondo sparring athlete. He is headed to Columbia University.

Kevin Seog led Oregon's top team in the Tests of Engineering Aptitude, Mathematics, and Science contest in 2020 and 2021. He is headed to Berkeley, where he plans to double major in business and computer science.

Kevin Minjay Seog

He co-led Oregons top team in the Tests of Engineering Aptitude, Mathematics, and Science contest this spring and last. He provided illustrations for a college-level economics book focusing on risk and insurance. He plays piano and guitar. He is headed to the University of California, Berkeley, where he intends to double major in computer science and business.

Stan Wing founded and led a nonprofit dedicated to providing free debate opportunities to middle schoolers across Oregon. He is headed to New York Universitys Stern School of Business to study business with concentrations in finance and econometrics.

Stanley Wang

During high school, he honed his skills as a landscape and portrait photographer. He competed as a member of Sunsets golf team. His primary extracurricular involvement during high school was with Health Occupations Students of America, an organization that holds state and international business competitions and conferences for high school students and its health careers counterpart. He helped found and served as president of the Oregon Middle School Speech and Debate League, a nonprofit dedicated to providing free debate opportunities to middle schoolers across Oregon. He is headed to New York Universitys Stern School of Business to study business with concentrations in finance and econometrics.

Lele Yang was a frequent volunteer during high school, including leading a group that taught computer science lessons to middle and high school students. She is headed to Penn State University, where she will take part in an accelerated premedical and medical program.

Lele Zixin Yang

Yang was president of Tech4Girls, in which she taught computer science lessons to middle and high school students. She gathered resources and grants for the program and also trained student teachers to help with the classes. She is an artist and a competitive fencer. Yang will enter the accelerated premedical-medical program at Penn State University in the fall.

Tualatin

Aven Sadighi, who competed in sports and business, is headed to Oregon State Universitys Honors College to study computer science with a focus on artificial intelligence.

Aven Sadighi

The Tualatin High valedictorian volunteered for three years tutoring fellow students. He competed and medaled at the 2020 Health Occupations Students of America state conference. He spent three years as a member of Pack Leaders, the student group that aids incoming freshmen in navigating their first year of high school. He competed freshman year in cross country, baseball and track and spent many hours volunteering in the community as a member of the National Honors Society. Hes headed to Oregon State Universitys Honors College to study computer science with a focus on artificial intelligence.

West Linn

Gillian Herbert

Herbert plans to study public policy in college, National Merit officials indicated.

Westview

Sangana Ilango

Ilango won a Congressional App Challenge for her mobile phone application, Spoiler Alert!, that helps consumers determine if food and medications are safe to use. She founded a club called CodeHERS to help encourage elementary girls to try computer coding. She worked as a research assistant at Oregon Health & Science University in summer 2019, interned at Mentor Graphics in summer 2020 and is interning with the product management team at Oregon Sesame this summer. Shes headed to the University of Southern California and will major in computer science.

Adam Jones

He conducted genomic research in Portland State Universitys Extreme Virus Lab. He competed at least one year for his high schools baseball, track and soccer teams. He is likely to study engineering in college, according to National Merit officials.

Pranav Mandyam, who placed in the top 10 in an international high school competition for biomedical laboratory science, is headed to Rice University to study cell biology and genetics.

Pranav Mandyam

He conducted research on acutemyeloidleukemia at Oregon Health & Science Universitys Knight Cancer Institute for two years. He was named one of the top 10 national finalists biomedical laboratory sciences by the Health Occupations Students of America. He was twice named a USA Biolympiadsemifinalist. He captained Westviews Science Olympiad Team, which made it to nationals. He volunteered at Legacy Good SamaritanHospital and received national-level recognition in multiple math competitions. He plays piano and received American College of Musicians high school diploma in social music. He is headed to Rice University, where he plans to major in biosciences with a concentration in cell biology and genetics.

Sophie Ong competed in speech and debate and did two years of research analyzing cancer genome databases and studying the impact of mRNA on tumors. At the University of California, Los Angeles, she plans to study genetics and related fields on a premed track.

Sophia Joy Ong

She competed as a member of Westviews speech and debate team. She served as co-president of Westviews chapter of Health Occupations Students of America and won that groups state championship in prepared speaking. She conducted research on cancer for two years at Oregon Health & Science University. She was also a captain of the school speech and debate team and competed at tournaments on both the state and national level. She is an avid guitar player and holds a third degree black belt in taekwondo. Shes headed to the University of California, Los Angeles, to study microbiology, immunology, and molecular genetics.

John Wang

He competed in and volunteered at many middle and high school robotics competitions, including serving as captain of his high school team. He competed as a member of Westviews varsity cross country and track teams. He was captain of the science club and president of the schools quiz bowl team, according to his profile on LinkedIn. He helped conduct research as an intern at Portland State Universitys Extreme Virus Lab. He is likely to study bioinformatics in college, National Merit officials said.

Note: If you know, or better are, one of these students, and I dont have a photo or full information, please contact me so I can make the coverage more complete.

-- Betsy Hammond; betsyhammond@oregonian.com; @chalkup

Excerpt from:
Half of Portland areas 22 top National Merit winners hail from just 2 schools - OregonLive

Imagine being able to reverse blindness, cure multiple sclerosis (MS), or rebuild your heart muscles after a heart attack. For the past few decades, research into stem cells, the building blocks of tissues and organs, has raised the prospect of medical advances of this kind yet it has produced relatively few approved treatments. But that could be about to change, says Robin Ali, professor of human molecular genetics of Kings College London. Just as gene therapy went from being a fantasy with little practical value to becoming a major area of treatment, stem cells are within a few years of reaching the medical mainstream. Whats more, developments in synthetic biology, the process of engineering and re-engineering cells, could make stem cells even more effective.

Stem cells are essentially the bodys raw material: basic cells from which all other cells with particular functions are generated. They are found in various organs and tissues, including the brain, blood, bone marrow and skin. The primary promise of adult stem cells lies in regenerative medicine, says Professor Ali.

Stem cells go through several rounds of division in order to produce specialist cells; a blood stem cell can be used to produce blood cells and skin stem cells can be used to produce skin cells. So in theory you can take adult stem cells from one person and transplant them into another person in order to promote the growth of new cells and tissue.

In practice, however, things have proved more complicated, since the number of stem cells in a persons body is relatively limited and they are hard to access. Scientists were also previously restricted by the fact that adult stem cells could only produce one specific type of cell (so blood stem cells couldnt produce skin cells, for instance).

In their quest for a universal stem cell, some scientists initially focused on stem cells from human embryos, but that remains a controversial method, not only because harvesting stem cells involves destroying the embryo, but also because there is a much higher risk of rejection of embryonic stem cells by the recipients immune system.

The good news is that in 2006 Japanese scientist Shinya Yamanaka of Kyoto University and his team discovered a technique for creating what they call induced pluripotent stem cells (iPSC). The research, for which they won a Nobel Prize in 2012, showed that you can rewind adult stem cells development process so that they became embryo-like stem cells. These cells can then be repurposed into any type of stem cells. So you could turn skin stem cells into iPSCs, which could in turn be turned into blood stem cells.

This major breakthrough has two main benefits. Firstly, because iPSCs are derived from adults, they dont come with the ethical problems associated with embryonic stem cells. Whats more, the risk of the body rejecting the cells is much lower as they come from another adult or are produced by the patient. In recent years scientists have refined this technique to the extent that we now have a recipe for making all types of cells, as well as a growing ability to multiply the number of stem cells, says Professor Ali.

Having the blueprint for manufacturing stem cells isnt quite enough on its own and several barriers remain, admits Professor Ali. For example, we still need to be able to manufacture large numbers of stem cells at a reasonable cost. Ensuring that the stem cells, once they are in the recipient, carry out their function of making new cells and tissue remains a work in progress. Finally, regulators are currently taking a hard line towards the technology, insisting on exhaustive testing and slowing research down.

The good news, Professor Ali believes, is that all these problems are not insurmountable as scientists get better at re-engineering adult cells (a process known as synthetic biology). The costs of manufacturing large numbers of stem cells are falling and this can only speed up as more companies invest in the area. There are also a finite number of different human antigens (the parts of the immune system that lead a body to reject a cell), so it should be possible to produce a bank of iPSC cells for the most popular antigen types.

While the attitude of regulators is harder to predict, Professor Ali is confident that it needs only one major breakthrough for the entire sector to secure a large amount of research from the top drug and biotech firms. Indeed, he believes that effective applications are likely in the next few years in areas where there are already established transplant procedures, such as blood transfusion, cartilage and corneas. The breakthrough may come in ophthalmology (the treatment of eye disorders) as you only need to stimulate the development of a relatively small number of cells to restore someones eyesight.

In addition to helping the body repair its own tissues and organs by creating new cells, adult stem cells can also indirectly aid regeneration by delivering other molecules and proteins to parts of the body where they are needed, says Ralph Kern, president and chief medical officer of biotechnology company BrainStorm Cell Therapeutics.

For example, BrainStorm has developed NurOwn, a cellular technology using peoples own cells to deliver neurotrophic factors (NTFs), proteins that can promote the repair of tissue in the nervous system. NurOwn works by modifying so-called Mesenchymal stem cells (MSCs) from a persons bone marrow. The re-transplanted mesenchymal stem cells can then deliver higher quantities of NTFs and other repair molecules.

At present BrainStorm is using its stem-cell therapy to focus on diseases of the brain and nervous system, such as amyotrophic lateral sclerosis (ALS, also known as Lou Gehrigs disease), MS and Huntingtons disease. The data from a recent final-stage trial suggests that the treatment may be able to halt the progression of ALS in those who have the early stage of the disease. Phase-two trial (the second of three stages of clinical trials) of the technique in MS patients also showed that those who underwent the treatment experienced an improvement in the functioning of their body.

Kern notes that MSCs are a particularly promising area of research. They are considered relatively safe, with few side effects, and can be frozen, which improves efficiency and drastically cuts down the amount of bone marrow that needs to be extracted from each patient.

Because the manufacture of MSC cells has become so efficient, NurOwn can be used to get years of therapy in one blood draw. Whats more, the cells can be reintroduced into patients bodies via a simple lumbar puncture into the spine, which can be done as an outpatient procedure, with no need for an overnight stay in hospital.

Kern emphasises that the rapid progress in our ability to modify cells is opening up new opportunities for using stem cells as a molecular delivery platform. Through taking advantage of the latest advances in the science of cellular therapies, BrainStorm is developing a technique to vary the molecules that its stem cells deliver so they can be more closely targeted to the particular condition being treated. BrainStorm is also trying to use smaller fragments of the modified cells, known as exosomes, in the hope that these can be more easily delivered and absorbed by the body and further improve its ability to avoid immune-system reactions to unrelated donors. One of BrainStorms most interesting projects is to use exosomes to repair the long-term lung damage from Covid-19, a particular problem for those with long Covid-19. Early preclinical trials show that modified exosomes delivered into the lungs of animals led to remarkable improvements in their condition. This included increasing the lungs oxygen capacity, reducing inflammation, and decreasing clotting.

Overall, while Kern admits that you cant say that stem cells are a cure for every condition, there is a lot of evidence that in many specific cases they have the potential to be the best option, with fewer side effects. With Americas Food and Drug Administration recently deciding to approve Biogens Alzheimers drug, Kern thinks that they have become much more open to approving products in diseases that are currently considered untreatable. As a result, he thinks that a significant number of adult stem-cell treatments will be approved within the next five to ten years.

Adult stem cells and synthetic biology arent just useful in treatments, says Dr Mark Kotter, CEO and founder of Bit Bio, a company spun out of Cambridge University. They are also set to revolutionise drug discovery. At present, companies start out by testing large numbers of different drug combinations in animals, before finding one that seems to be most effective. They then start a process of clinical trials with humans to test whether the drug is safe, followed by an analysis to see whether it has any effects.

Not only is this process extremely lengthy, but it is also inefficient, because human and animal biology, while similar in many respects, can differ greatly for many conditions. Many drugs that seem promising in animals end up being rejected when they are used on humans. This leads to a high failure rate. Indeed, when you take the failures into account, it has been estimated that it may cost as much to around $2bn to develop the typical drug.

As a result, pharma companies are now realising that you have to insert the human element at a pre-clinical stage by at least using human tissues, says Kotter. The problem is that until recently such tissues were scarce, since they were only available from biopsies or surgery. However, by using synthetic biology to transform adult stem cells from the skin or other parts of the body into other types of stem cells, researchers can potentially grow their own cells, or even whole tissues, in the laboratory, allowing them to integrate the human element at a much earlier stage.

Kotter has direct experience of this himself. He originally spent several decades studying the brain. However, because he had to rely on animal tissue for much of his research he became frustrated that he was turning into a rat doctor.

And when it came to the brain, the differences between human and rat biology were particularly stark. In fact, some human conditions, such as Alzheimers, dont even naturally appear in rodents, so researchers typically use mice and rats engineered to develop something that looks like Alzheimers. But even this isnt a completely accurate representation of what happens in humans.

As a result of his frustration, Kotter sought a way to create human tissues. It initially took six months. However, his company, Bit Bio, managed to cut costs and greatly accelerate the process. The companys technology now allows it to grow tissues in the laboratory in a matter of days, on an industrial scale. Whats more, the tissues can also be designed not just for particular conditions, such as dementia and Huntingdons disease, but also for particular sub-types of diseases.

Kotter and Bit Bio are currently working with Charles River Laboratories, a global company that has been involved in around 80% of drugs approved by the US Food and Drug Administration over the last three years, to commercialise this product. They have already attracted interest from some of the ten largest drug companies in the world, who believe that it will not only reduce the chances of failure, but also speed up development. Early estimates suggest that the process could double the chance of a successful trial, effectively cutting the cost of each approved drug by around 50% from $2bn to just $1bn. This in turn could increase the number of successful drugs on the market.

Two years ago my colleague Dr Mike Tubbs tipped Fate Therapeutics (Nasdaq: FATE). Since then, the share price has soared by 280%, thanks to growing interest from other drug companies (such as Janssen Biotech and ONO Pharmaceutical) in its cancer treatments involving genetically modified iPSCs.

Fate has no fewer than seven iPSC-derived treatments undergoing trials, with several more in the pre-clinical stage. While it is still losing money, it has over $790m cash on hand, which should be more than enough to support it while it develops its drugs.

As mentioned in the main story, the American-Israeli biotechnology company BrainStorm Cell Therapeutics (Nasdaq: BCLI) is developing treatments that aim to use stem cells as a delivery mechanism for proteins. While the phase-three trial (the final stage of clinical trials) of its proprietary NurOwn system for treatment of Amyotrophic lateral sclerosis (ALS, or Lou Gehrigs disease) did not fully succeed, promising results for those in the early stages of the disease mean that the company is thinking about running a new trial aimed at those patients. It also has an ongoing phase-two trial for those with MS, a phase-one trial in Alzheimers patients, as well as various preclinical programmes aimed at Parkinsons, Huntingtons, autistic spectrum disorder and peripheral nerve injury. Like Fate Therapeutics, BrainStorm is currently unprofitable.

Australian biotechnology company Mesoblast (Nasdaq: MESO) takes mesenchymal stem cells from the patient and modifies them so that they can absorb proteins that promote tissue repair and regeneration. At present Mesoblast is working with larger drug and biotech companies, including Novartis, to develop this technique for conditions ranging from heart disease to Covid-19. Several of these projects are close to being completed.

While the US Food and Drug Administration (FDA) controversially rejected Mesoblasts treatment remestemcel-L for use in children who have suffered from reactions to bone-marrow transplants against the advice of the Food and Drug Administrations own advisory committee the firm is confident that the FDA will eventually change its mind.

One stem-cell company that has already reached profitability is Vericel (Nasdaq: VCEL). Vericels flagship MACI products use adult stem cells taken from the patient to grow replacement cartilage, which can then be re-transplanted into the patient, speeding up their recovery from knee injuries. It has also developed a skin replacement based on skin stem cells.

While earnings remain relatively small, Vericel expects profitability to soar fivefold over the next year alone as the company starts to benefit from economies of scale and runs further trials to expand the range of patients who can benefit.

British micro-cap biotech ReNeuron (Aim: RENE) is developing adult stem-cell treatments for several conditions. It is currently carrying out clinical trials for patients with retinal degeneration and those recovering from the effects of having a stroke. ReNeuron has also developed its own induced pluripotent stem cell (iPSC) platform for research purposes and is seeking collaborations with other drug and biotech companies.

Like other small biotech firms in this area, it is not making any money, so it is an extremely risky investment although the rewards could be huge if any of its treatments show positive results from their clinical trials.

More here:
Investing in stem cells, the building blocks of the body - MoneyWeek

Newswise Irvine, CA May 13, 2021 A new University of California, Irvine-led study finds low serum levels of the sugar N-acetylglucosamine (GlcNAc), is associated with progressive disability and neurodegeneration in multiple sclerosis (MS).

The study, done in collaboration with researchers from Charit Universittsmedizin Berlin, Germany, and the University of Toronto, Canada, is titled, Association of a Marker of N-Acetylglucosamine With Progressive Multiple Sclerosis and Neurodegeneration, The study was published this week in JAMA Neurology.

The study suggests that GlcNAc, which has been previously shown to promote re-myelination and suppress neurodegeneration in animal models of MS, is reduced in serum of progressive MS patients and those with worse clinical disability and neurodegeneration.

We found the serum levels of a marker of GlcNAc was markedly reduced in progressive MS patients compared to healthy controls and patients with relapsing-remitting multiple sclerosis explained Michael Demetriou, MD, PhD, FRCP(C), professor of neurology, microbiology and molecular genetics at UCI School of Medicine, and senior author on the paper.

First author of the study, Alexander Brandt, MD, adjunct associate professor of neurology at the UCI School of Medicine and previously associated with the Experimental and Clinical Research Center, Charit Universittsmedizin Berlin and Max Delbrueck Center for Molecular Medicine, Germany, added, Lower GlcNAc serum marker levels correlated with multiple measures of neurodegeneration in MS, namely worse expanded disability status scale scores, lower thalamic volume, and thinner retinal nerve fiber layer. Also, low baseline serum levels correlated with a greater percentage of brain volume loss at 18 months, he said.

GlcNAc regulates protein glycosylation, a fundamental process that decorates the surface of all cells with complex sugars. Previous preclinical, human genetic and ex vivo human mechanistic studies revealed that GlcNAc reduces proinflammatory immune responses, promotes myelin repair, and decreases neurodegeneration. Combined with the new findings, the data suggest that GlcNAc deficiency may promote progressive disease and neurodegeneration in patients with MS. However, additional human clinical studies are required to confirm this hypothesis.

Our findings open new potential avenues to identify patients at risk of disease progression and neurodegeneration, so clinicians can develop and adjust therapies accordingly, said Michael Sy, MD, PhD, assistant professor in residence in the Department of Neurology at UCI and a co-author of the study.

MS is characterized by recurrent episodes of neurologic dysfunction resulting from acute inflammatory demyelination. Progressive MS is distinguished by continuous inflammation, failure to remyelinate, and progressive neurodegeneration, causing accrual of irreversible neurologic disability. Neurodegeneration is the major contributor to progressive neurological disability in MS patients, yet mechanisms are poorly understood and there are no current treatments for neurodegeneration.

This study was funded in part by a grant from the National Institute of Allergy and Infectious Disease and the National Center for Complimentary and Integrative Health as well as the Excellence Initiative and the Excellence Strategy of the German Federal and State Governments.

About the UCI School of Medicine

Each year, the UCI School of Medicine educates more than 400 medical students, and nearly 150 doctoral and masters students. More than 700 residents and fellows are trained at UCI Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and masters degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/masters in public health, or an MD/masters degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.

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New study finds low levels of a sugar metabolite associates with disability and neurodegeneration in multiple sclerosis - Newswise

Sara Cernadas-Martn with the research vessel Seawolf behind her. Photo by John Griffin

Sara Cernadas-Martn is a self-described interdisciplinary scientist with knowledge spanning the fields of marine biology, molecular genetics, and conservation ecology, with an M.S. and a PhD from Stony Brook University.

Beyond her passion for studying and preserving the diversity of marine life in our local waters, Cernadas-Martn is equally as dedicated to fostering diversity within the human race, making her the ideal person to serve as co-chair for the School of Marine and Atmospheric Science (SoMAS) Diversity, Equity and Inclusivity Committee.

As an Hispanic scientist, I am committed to increasing diversity on campus and making an extra effort to promote higher education within the Hispanic community and other underrepresented racial, ethnic and social groups, Cernadas-Martn said.Hispanics are highly underrepresented in undergraduate and graduate schools in America, which is especially discouraging when considering that the Hispanic population is the largest ethnic minority group in the country. I strongly believe that having a diverse student body benefits everyone involved.

For her masters project, Sara studied the distribution of ammonia oxidizing bacteria in the Cariaco Basin near Venezuela, an oxygen minimum zone with a permanently anoxic (oxygen-depleted) deep layer, under the supervision of Professor Gordon Taylor. Her PhD thesis focused on the multidisciplinary ecological characterization of summer flounder (Paralichthys dentatus) in Shinnecock Bay using acoustic telemetry, diet analysis and otolith (earstones in bony fishes) microchemistry under the supervision of Professor Ellen Pikitch.

Cernadas-Martn is currently working with the Institute for Ocean Conservation Science (IOCS) as a senior postdoctoral associate at Stony Brook, where her work focuses on managing the research component of ShiRP fisheries (Shinnecock Bay Restoration Program) and establishing a new environmental DNA program for tracking fish species richness in the South Shore Long Island estuaries.

In 2019, Cernadas-Martn was also selected as the recipient of the distinguished Nuria Protopopescu Memorial Teaching Award, presented annually to a graduate student based on demonstrated excellence in teaching, innovation and creativity in instructional plans and materials, and engagement with and dedication to their students.

Although I enjoy working with students of all backgrounds, I have always taken special interest in Latinx students, making sure they felt motivated, included and most importantly, had fun while doing science, she said. At first, many of these students were hesitant to be assertive while in the field or in the classroom. I came to realize, in sharing the same language, I could help students to overcome their natural timidity and become more engaged in their research and learning experience.

Of particular interest to Cernadas-Martn is extending educational opportunities to students from diverse backgrounds, including first-generation college students, international students, and students with a wide range of educational experiences and goals.

Her academic achievements and community involvement, along with her focused effort on student diversity and integration, have been recognized by Stony Brook University with the prestigious W. Burghardt Turner Fellowship, a highly competitive fellowship which acknowledges the academic and research achievements of underrepresented doctoral students and requires a strong commitment to inclusivity and community development.

Another of Cernadas-Martns personal goals is to help improve the integration of international students into the wider academic community.

I remember when I first got to Stony Brook University from Spain. I was in shock, she recalled. I was happy for having made it into graduate school, but at the same time had to deal with a language barrier and being away from my family and friends. The first few months are critical times in the life of an international student and can potentially handicap academic performance.

Cernadas-Martn believes efficient integration is key to helping international students realize that there are opportunities in this new chapter of their lives. During my first years of graduate school, I did my best to help improve social interactions among students in my department, she said. One example were the flamenco nights I hosted once a month at a Spanish restaurant open to everyone in my department, where I was able to share the culinary, musical and folkloric traditions of my culture.

To that end, Cernadas-Martn has volunteered with the Graduate Student Club of her department as the activities coordinator, creating and running a departmental photo competition since 2012, among other undertakings.

Perhaps some of her compassion originated from her own medical struggles.As a college freshman, she overcame bone cancer, which impeded her from attending school. She recovered, but suffered a relapse in the middle of her sophomore year.

During my graduate career I have been, once again, plagued with health issues, which included three major spinal surgeries, she said, adding that the surgeries set back her graduation timeline and consequently constrained the ability of her research grants to cover laboratory costs and living expenses.

However, I was lucky enough to have a few professors, family and friends who supported me and encouraged me to finish my degree, she said. It did take me longer than most people, but I did it. Looking back, I cant thank those people in my life enough. I aim to be a support system for as many students as possible, motivating them to move forward despite setbacks and encouraging them to pursue their passion.

Glenn Jochum

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Cernadas-Martn Is a Champion for Marine and Human Diversity | | SBU News - Stony Brook News

Four Penn Faculty: Election to the National Academy of Sciences

Four members of the University of Pennsylvania faculty have been elected to the United States National Academy of Sciences (NAS). They join 120 members, 59 of whom are women, the most elected in a single year, and 30 international members, elected by their peers this year to NAS. Recognized for distinguished and continuing achievements in original research, this new class brings the total number of active members to 2,461 and of international members to 511.

Marisa Bartolomei is the Perelman Professor of Cell and Developmental Biology in the department of cell and developmental biology in the Perelman School of Medicine. She is also the co-director of the Penn Epigenetics Institute. Crossing into the disciplines of cell and molecular biology, pharmacology, and neuroscience, Dr. Bartolomei and her lab investigate genomic imprinting in mice. Specifically, they focus on the H19 gene, which is only expressed in maternal alleles, in order to better uncover the mechanisms behind imprinting and the effects of the environment, assisted reproductive technologies, and endocrine disruptors. Her lab also looks into the molecular and genetic systems behind X inactivation in mice. Her research has been published widely in journals including Nature, Nature Biotechnology, Development, and PLoS Genetics.

Michael Kearns is the National Center Professor of Management & Technology in the department of computer and information science in the School of Engineering and Applied Science. He also holds secondary appointments in the School of Arts & Sciences department of economics and the departments of statistics and operations, information and decisions at the Wharton School. He is an expert in machine learning, algorithmic game theory, and microeconomics, and applies both theoretical research and experimental techniques to better understand the social dimensions of new information technology, such as its impact on privacy and fairness. Dr. Kearns is also the founding director of Penns Warren Center for Network and Data Sciences, which draws on researchers from around the University to study some of the most pressing problems of the digital age. Dr. Kearns is also the co-author of The Ethical Algorithm, which shows how seemingly objective data science techniques can produce biased outcomes.

Diana Mutz is the Samuel A. Stouffer Professor of Political Science and Communication in the Annenberg School for Communication. She also serves as director of the Institute for the Study of Citizens and Politics; she is also an affiliate of the Warren Center. She studies political communication, political psychology, and public opinion, and her research focuses on how the American mass public relates to the political world and how people form opinions on issues and candidates. She received a 2017 Carnegie Fellowship and a 2016 Guggenheim Fellowship to pursue research on globalization and public opinion, and in 2011 received the Lifetime Career Achievement Award in Political Communication from the American Political Science Association. In addition to many journal articles, Dr. Mutz is the author of Impersonal Influence: How Perceptions of Mass Collectives Affect Political Attitudes, Hearing the Other Side: Deliberative Versus Participatory Democracy, and In-Your-Face Politics: The Consequences of Uncivil Media.

M. Celeste Simon is the Arthur H. Rubenstein, MB BCh, Professor in the department of cell and developmental biology in the Perelman School of Medicine and the scientific director of The Abramson Family Cancer Research Institute. She and her lab research the metabolism of cancer cells, tumor immunology, metastasis, and how healthy cells and cancer cells respond to a lack of oxygen and nutrients. Her work uses both animal models and cancer patient samples, and her goal is to create techniques to treat various tumors like kidney cancer, soft tissue sarcoma, liver cancer, and pancreatic cancer. Dr. Simon was the recipient of a National Cancer Institute Outstanding Investigator Award in 2017, and she has authored more than 275 articles in journals including Cell, Science, Nature, Cancer Discovery, Nature Genetics, and Cancer Cell.

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Four Penn Faculty: Election to the National Academy of Sciences - UPENN Almanac

The controversy over genetically engineered organisms (sometimes called genetically modified organisms, or GMOs) is genuine, not faux but only because of uninformed, exaggerated concerns about the most recent techniques newness. What is faux and disingenuous are the arguments put forth by genetic engineerings opponents.

Humans have been modifying the DNA of our food for thousands of years (even though we didnt know that DNA was mediating the changes until the 20th century).We call it agriculture.Early farmers (>10,000 years ago) used selective breeding to guide DNA changes in crops and animals to better suit our needs.Approximately a hundred years ago plant breeders began using harsh chemicals and/or radiation to randomly change, or mutate, the DNA of crop plants.These mutagens caused innumerable changes to the DNA, none of which was characterized or examined for safety.The plant breeders looked and selected for desired traits of various kinds.Problems were rare.

Today more than half of all food crops have mutagenesis breeding as part of their pedigree. Ancestral varieties bear little resemblance to the domesticated crops we eat today. There are many striking pictorial examples here.

Approximately 40 years ago agricultural scientists and plant breeders began to use recombinant DNA technology (gene splicing) to make far more precise and predictable changes to the DNA in our crop plants.This molecular genetic engineering (GE) typically takes a gene with a known function, e.g., one that expresses a protein toxic to certain insect predators, and transfers it into a crop, enabling the GE crop to protect itself from insect pests. This one trait, resulting from the introduction of a gene from the bacterium Bacillus thuringiensis (abbreviated Bt) into plants, has allowed farmers around the world to reduce broad spectrum insecticide spraying by billions of pounds.

Since the advent in the 1970s of this recombinant DNA technology, which enables segments of DNA to be moved readily and more precisely from one organism to another, molecular genetic engineering techniques have become increasingly more sophisticated, precise, and predictable. This evolution has culminated in the most recent discoveries, the CRISPR-Cas9 system and base editing.

CRISPR (short for Clustered Regularly Interspaced Short Palindromic Repeats) is a natural defense system that bacteria use against invading viruses. CRISPR can recognize specific DNA sequences, while the enzyme Cas9 cuts the DNA at the recognized sequence. As often happens in scienceand reminiscent of mutagenesis a century ago and recombinant DNA technology in the 1970smolecular biologists quickly copied, adapted, and improved the naturally occurring system. Using CRISPR-Cas9, scientists can target and edit DNA at precise locations, deleting, inserting, or modifying genes in microorganisms, plants and animals, and even humans.

CRISPR-Cas9 presages a revolution in agriculture and human medicine because it is so much more precise and predictable than earlier techniques. Precision and predictability are important to ensure that results are safe and achieve their desired ends. There are notable historical examples of older, pre-molecular techniques of genetic modification in agriculture that misfired.Examples include:

Despite their success for farmers of all types, from subsistence to huge-scale commercial, GE crops have been discriminated against by regulators and demonized by activists. In the early 1970s, at a conclave now referred to as the Asilomar Conference, a group of scientists none involved in agriculture or food science raised concerns about hypothetical hazards that might arise from the use of the newly discovered molecular genetic modification technique recombinant DNA technology, or gene-splicing. However, they failed to appreciate the history of genetic modification by means of cruder, less predictable technologies, described below.

The Asilomar Conference led to guidelines published by the U.S. National Institutes of Health (NIH) for the application of these techniques for any purpose.These process-based guidelines, which were applicable exclusively to recombinant DNA technology, were in addition to the preexisting product-focused regulatory requirements of other federal agencies that had statutory oversight of food, drugs, certain plants, pesticides, and so on.

The NIH guidelines, which were in effect the original sin of precautionary, unscientific regulation, were quite stringent. For example, without regulatory approval, the intentional release of recombinant DNA-modified organisms into the environment, or fermentation (in contained fermenters) at volumes greater than ten liters, required explicit prior approval by the NIH and local Institutional Biosafety Committees.

Given the seamless continuum of techniques for genetic modification described above, such requirements were unwarranted.No analogous blanket restrictions existed for similar or even virtually identical plants, microorganisms, or other organisms modified by traditional techniques, such as chemical or irradiation mutagenesis or wide-cross hybridizations.

Thus, uninformed, ill-founded, and exaggerated concerns about the risks of recombinant DNA-modified organisms in medical, agricultural, and environmental applications precipitated the regulation of recombinant organismsregulation triggered simply by the process, or technique, of genetic modification, rather than the product, i.e., the characteristics of the modified organism itself. This was an unfortunate precedentas was entrusting technology regulation to a research agency, the NIH, whose legacy plagues regulation worldwide today. Most industrial countries, including the US, have specific regulatory agencies like the US Food and Drug Administration and European Medicines Agency that regulate product safety. Research agencies rarely are involved in regulating products or processes.

The regulatory burden on the use of recombinant DNA technologywhich some people (mostly activists and regulators) consider gives rise to a mythical category of organisms called Genetically Modified Organisms, or GMOs, was, and remains, disproportionate to its risk, and the opportunity costs of regulatory delays and expenses are formidable. According to Wendelyn Jones at DuPont Crop Protection, a survey found that the cost of discovery, development and authorization of a new plant biotechnology trait introduced between 2008 and 2012 was $136 million. On average, about 26 percent of those costs ($35.1 million) were incurred as part of the regulatory testing and registration process.

A salient question currently for regulators, scientists, and consumers is whether gene editing will fall down the same rabbit hole. Unfortunately, much of the discussion focuses on irrelevant issues such as whether organisms that could arise naturally or that are not transgenic (containing DNA from different sources) should be subject to more lenient regulation than GMOs. As should be evident from the discussion above, such issues have no implications for riskand, therefore, for regulation. In fact, modern plant breeding techniques, including genome editing, are more precise, circumscribed, and predictable than other methods in other words, if anything, likely to be safer. This assessment is neither new nor novel. A landmark report from the U.S. National Research Council concluded in 1989:

These critical points, clearly articulated more than 30 years ago and about which there is virtual unanimity in the scientific community, have not sunk in.

There is an ongoing need for genetic modification in agriculture. Gene editing could play a key role in Englands sugar beet sector, for example, and Britains farming and in February, environment minister George Eustice told the annual conference of the National Farmers Union that the sugar beet sector could use the assistance of gene editing technologies to overcome yield reduction due to virus infection. He added: Gene editing is really just a more targeted, faster approach to move traits from one plant to another but within the same species so in that respect it is no different from conventional breeding.

The first part of Eustaces statement is accurate, but the second part gives the misimpression that although gene edited crops are analogous to conventional breedingand, therefore, presumably harmlessthey are sufficiently far removed from dreaded GMOs that they should be exempt from the onerous regulation appropriate for the latter. Until now, in the European Union, gene editing has been strictly regulated in the same way as GMOs. Their oversight might diverge in the future, however, inasmuch as serious attention is being paid to this new technology and its enormous potential.

But preferential regulatory treatment of gene editing over recombinant DNA-mediated modifications would represent expediency over logic: The NRC report (as well as other, innumerable, similar analyses) makes it clear that an approach that deregulates gene editing but not recombinant DNA modifications would ignore the seamless continuum that exists among methods of genetic modification, and that it would be unscientific.There is no reason to throw transgenic recombinant DNA constructions under the regulatory bus.

The relationships among genome editing, plant breeding, and GMO crops are more interconnected, complex and nuanced than it may appear at first glance.Plant breeding itself has long been a murky science in terms of genetics and heredity.While Britains Eustice lauds genome editing because it involves only intra-species modification, the history of plant breeding has long included distant or wide crosses to move beneficial traits such as disease resistance from one plant species or one genus to another.Almost a century of wide cross hybridizations, which involve the movement of genes from one species or genus to another, has given rise to plantsincluding everyday varieties of corn, oats, pumpkin, wheat, black currants, tomatoes, and potatoes, among othersthat do not and could not exist in nature. Indeed, with the exception of wild berries, wild game, wild mushrooms, and fish and shellfish, virtually everything in North American and European diets has been genetically improved in some way.Compared to the new molecular modification technologies, these wide crosses are crude and less predictable.

Another wrinkle is that plant scientists have discovered what have been termed natural GMOs, which further confounds the terminology.These include whiteflies harboring plant genes that protect them from pesticides, horizontal gene transfer between different species of grasses, sweet potato harboring sequences from the bacterium Agrobacterium, and aphids which express a red fungal pigment to protect them from would-be predators.This is more evidence that the term GMO itself has become meaningless.

This brings us back to the regulatory conundrum surrounding the way forward with the various products of genetic engineering using different technologies. Eager to avoid the delays, impasses and rejections and inflated opportunity costs that have confounded GMOs, many in the scientific and commercial communities are willing to play down the novelty of genome editing, while, in effect, conceding that recombinant DNA constructions should continue to be stringently regulated.

However, as we have discussed, the comparison of genome editing and recombinant DNA is a distinction without a difference, especially when viewed against the backdrop of the crude constructions of (largely unregulated) traditional plant breeding.Trying to draw meaningful distinctions between molecular genetic engineering and other techniques for the purpose of regulation is rather like debating how many angels can dance on the head of a pin.Its way past time that for purposes of regulatory policy, we began to think in terms of the risk posed by organisms and their products, rather than which technology(ies) was employed.

Kathleen Hefferon, Ph.D., teaches microbiology at Cornell University. Find Kathleen on Twitter@KHefferon

Henry Miller, a physician and molecular biologist, is a senior fellow at the Pacific Research Institute. He was a Research Associate at the NIH and the founding director of the U.S. FDAs Office of Biotechnology. Find Henry on Twitter@henryimiller

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Is there a difference between a gene-edited organism and a 'GMO'? The question has important implications for regulation - Genetic Literacy Project

This year, five Wesleyan students were inducted into the American Society for Biochemistry and Molecular Biology (ASBMB) Honor Society. Thirty-one students nationwide were given this honor.

Inducted students must be juniors or seniors with a GPA of 3.4 or higher on a 4.0 scale, belong to a student chapter of the ASBMB, and demonstrate exceptional achievement in academics, undergraduate research and science outreach, according to the website.

The inducted students include the following:

Nour-Sada Harzallah 21, a College of Integrative Sciences student majoring in molecular biology & biochemistry (MB&B) and physics. Harzallah, from Tunisia, works in Professor Francis Starrs physics lab, belongs to the Wesleyan Women in Science steering committee, and is a STEM intern for the Office of Equity and Inclusion.

Her involvement in racial and gender equity in STEM has shaped her commitment to work on projects that serve the underrepresented and marginalized outside the lab and from the lab bench, reads her ASBMB bio. Her wildest dream is to develop initiatives that translate cutting-edge technologies into accessible and marketable means of diagnosis and therapeutics in her home country of Tunisia.

Jack Kwon 21, who works with Professor of Biology Michael Weir to study the ribosome.

We are aiming to elucidate the function of a highly conserved region of the ribosome called the CAR interaction surface through wet lab experiments and dry lab Molecular Dynamics simulations, Kwon wrote in his ASBMB bio. Kwon intends to graduate with a masters degree in MB&B through Wesleyans BA/MA program before pursuing a PhD in a related field.

Shawn Lin 22, who is majoring in biology, MB&B, and biophysics. Lin works in the MB&B lab of Professor Ishita Mukerji and the physics lab of Professor Candice Etson.

His research topic is Elucidation of interactions between integration host factor and a DNA four-way junction, reads Lins ASBMB bio. In addition to research, he is also the founder of NORDSAC (National Organization for Rare Disorders Student Association Connecticut). The goal of this organization is to raise awareness of rare disorders among students in Connecticut through fundraising, guest lectures, and rare disease day events.

Alex Poppel, a masters student in the MB&B department. Poppell works in Professor Amy MacQueens MB&B lab.

As a member of Wesleyans ASBMB Student Chapter, his outreach involvement has mainly focused on improving his schools community, such as by promoting undergraduate research opportunity awareness and equity and inclusion efforts in the sciences, Poppels ASBMB bio reads.

Maya Vaishnaw 21, a double major in psychology and MB&B. Vaishnaw works with Professor Erika Taylor in her chemistry lab.

The Taylor Lab takes a multidisciplinary approach to characterizing enzymes with applied chemical and biomedicinal applications, Vaishnaws ASBMB bio reads. In the future, Maya hopes to pursue research in clinical genetics.

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5 Students Inducted Into American Society for Biochemistry and Molecular Biology Honor Society - Wesleyan Connection

All cellular life on Earth is based on DNA, which transfers informationabout everything from hair color to personality traitsfrom one generation to the next. The four chemical bases that convey this information are adenine (A), cytosine (C), guanine (G), and thymine (T).

The other essential information molecule on Earth is RNA, in which thymine (T) is replaced by uracil (U). RNA has a one-string structure rather than a double-string structure like DNA. The first cellular life on our planet is thought to have relied exclusively on this means of transferring genetic informationin the so-called RNA worldand even today there are viruses (like the one that causes COVID) that only use RNA.

In a paper recently published in Science, a research group led by Dona Sleiman from the Institute Pasteur in Paris has discovered that some viruses show more variation in their genetic coding than was previously known. In the RNA of these viruses, adenine (A) is replaced with Z, where Z stands for diaminopurine.

This follows an earlier study by Zunyi Yang and colleagues at the Foundation for Applied Molecular Evolution in Gainesville, Florida, showing that an artificial genetic system could be created by adding two additional non-standard bases to ordinary DNA. Amazingly, the artificial six-base system continued to evolve rather than reverting back to the natural four-base system. This implies that the DNA we take as standardmade of A, C, G, and Tis just one of many viable solutions to the challenge of biological information transfer.

The variability does not stop here. Strings of DNA are organized in base triplets that determine which of the standard 20 amino acids are assigned to synthesize proteins. However, these triplet assignments are not universal. For example, CUG, which usually codes for the amino acid serine, instead codes for the amino acid leucine in some types of fungi. Also, some organisms naturally encode for two additional amino acids instead of the standard 20 amino acids.

What does this brief excursion into genetics have to do with alien life? While it is believed that all life on our planet derives from one common ancestor, the genetic code is much more flexible and diverse than usually appreciated. The biochemistry of information transfer in an alien species would almost certainly use different building blocks and encodings, and perhaps even a different number of bases. Our genetic code is surely highly optimized for life on Earth, but I feel certain that there are many optimal solutionsperhaps some that are even betterfor transferring information chemically from one generation to the next.

We, of course, cannot say what type of genetic code an alien species would use. But given that it would most likely be biochemically different, it would mostly likely be easily distinguishable from life on Earth. It may even be more different than we expect. A fascinating out-of-the-box genetic system has been suggested by Gerald Feinberg and Robert Shapiro, based on magnetic orientations rather than chemistry. They showed how magnetized particles, when approaching a magnetic chain, will align with the chain. As a result, the chain is duplicated, and this method could in principle be used to convey information in a binary code.

So, while alien life may well transmit genetic information using structures similar to RNA and DNA, we should always be prepared to expect the unexpected.

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The Science of Aliens, Part 2: What Kind of Genetic Code Would Extraterrestrials Have? - Air & Space Magazine

A UT Austin faculty member, Dr. Shelley Marshall Payne, has been named a recipient of the prestigious Piper Professor Award, an annual award that recognizes outstanding college professors from colleges and universities across the state of Texas.

Dr. Shelley Marshall Payne is a professor in the department of Medical Education and Molecular Biosciences housed in the College of Natural Sciences. Her research interests are in genetics and regulation of virulence factors of gram negative pathogens, including Shigella and Vibrio cholerae.

We are so proud of Dr. Payne and thrilled to see her recognized for her superior teaching and advancement of our students learning during what has been an especially challenging year for all, said senior vice provost of faculty affairs, Tasha Beretvas. We are also very grateful to the Minnie Stevens Piper Foundation for their support for teaching excellence through these awards. And we acknowledge the honor that one of our faculty members receiving this award reflects on The University of Texas at Austin.

The Piper Professor Award was established by the San Antoniobased Minnie Stevens Piper Foundation in 1958 and honors 10 professors per academic year for their dedication to the teaching profession and for their outstanding academic, scientific and scholarly achievement. Each Piper Professor receives a certificate of merit, a gold pin and a $5,000 honorarium.

Selection is made on the basis of nominations; each two and four-year college and university in the state may submit only one nominee annually.

More information is available on the Texas Comptroller website.

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UT Austin Faculty Member Receives 2021 Piper Professor Award - Office of the Executive Vice President and Provost - UT News | The University of Texas...

One of the University's leading scientists, Professor Vernonica (Noni) Franklin-Tong has been elected a Fellow of the Royal Society.

Professor Franklin-Tong, a plant scientist, was recognised for her pioneering work on self-incompatibility in plants the mechanism which prevents plants from inbreeding. Using the common field poppy as a model system, she has identified novel mechanisms pivotal to regulation of cell growth and programmed cell death in plants.

She joins more than 60 outstanding scientists from around the globe who have been elected to the Royal Society this year as Fellows and Foreign Members.

Announcing the new appointments, Sir Adrian Smith, President of the Royal Society, said: The global pandemic has demonstrated the continuing importance of scientific thinking and collaboration across borders. Each Fellow and Foreign Member bring their area of scientific expertise to the Royal Society and when combined, this expertise supports the use of science for the benefit of humanity.

Our new Fellows and Foreign Members are all at the forefronts of their fields from molecular genetics and cancer research to tropical open ecosystems and radar technology. It is an absolute pleasure and honour to have them join us.

Professor Franklin-Tong said: "I am absolutely delighted to be elected as a Fellow of The Royal Society. It's a huge honour to have this recognition and to join this prestigious group of scientists. I'm indebted to the contribution of my team of researchers over the years.

"As a female professor of mixed ethnicity, I am especially proud of this achievement. I hope it inspires others to reach for the seemingly impossible."

Dr Neil Hotchin, Head of the School of Biosciences, said: Noni is an outstanding plant biologist and her election to the Royal Society is a well-deserved recognition of her truly ground-breakingwork on self-incompatibility in plantswhich has resultedin multiple high impactpapers in journals such asScience and Nature.

For media enquiries please contact Beck Lockwood, Press Office, University of Birmingham, tel: +44 (0)781 3343348.

The University of Birmingham is ranked amongst the worlds top 100 institutions. Its work brings people from across the world to Birmingham, including researchers, teachers and more than 6,500 international students from over 150 countries.

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Distinguished University of Birmingham plant scientist elected to the Royal Society - University of Birmingham

When you pair graduate students with undergraduates, what do they talk about? In the case of the University of Virginias Double Hoo Awards, they discuss interactive machine learning, the genesis of false memories, how the brain controls infection and politicization in the field of intelligence, among other things.

This year, UVA awarded 21 Double Hoo Awards to new pairings of undergraduates and graduate student mentors, with the teams receiving up to $6,000. One team from last years recipients was awarded a renewal to continue their research, receiving $3,000 in support. The Double Hoo Award is funded by the Robert C. Taylor Fund.

We love seeing the ways that these student pairs have come together to pursue research and creative inquiry, Andrus G. Ashoo, director of the Office of Undergraduate Research, said. The funded projects represent disciplines across the institution and are pursuing some fascinating questions. It gets me excited for next years research symposium, where they will all present.

While undergraduate research is typically done in close collaboration with faculty members, the Double Hoo Awards add another element: the involvement of a graduate student mentor who plays a key role in defining the project. In the Double Hoo process, the undergraduate student submits the application with a project proposal and budget and identifies a graduate student with whom he or she will work. The graduate student also submits a statement of mentorship as part of the application process.

Not only will the research these students pursue be valuable to their development intellectually, it will also help these students professionally and socially as they learn to navigate a new relational dynamic, Ashoo said. In addition, an opportunity like this can be an experience that helps to clarify questions that the undergraduate or graduate might have about their future goals. For the graduate students, this is an invaluable opportunity to develop as a mentor, learning to provide supervision and incorporating the undergraduate into the larger project goals. This experience will be important, whether they go on to roles in academia, industry or public service.

This years Double Hoo recipients are:

One of last years projects has been renewed for a second year:

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Double Hoo Research: Undergrads and Grads Team Up to Create Knowledge - University of Virginia

Global Genetic Testing Market research report has been prepared with a nice combination of industry insight, smart solutions, practical solutions and newest technology to give better user experience. Under market segmentation chapter, research and analysis is done based on several market and industry segments such as application, vertical, deployment model, end user, and geography. To perform this market research study, competent and advanced tools and techniques have been utilized that include SWOT analysis and Porters Five Forces Analysis. Businesses can surely anticipate the reduced risk and failure with the winning research report.

Global Genetic Testing Market, By Type (Predictive & Presymptomatic Testing, Carrier Testing, Prenatal & Newborn Testing, Diagnostic Testing, Pharmacogenomic Testing, Others), Technology (Cytogenetic Testing, Biochemical Testing, and Molecular Testing), Application (Cancer Diagnosis, Genetic Disease Diagnosis, Cardiovascular Disease Diagnosis, Others), Disease (Alzheimers Disease, Cancer, Cystic Fibrosis, Sickle Cell Anemia, Duchenne Muscular Dystrophy, Thalassemia, Huntingtons Disease, Rare Diseases, Other Diseases), Product (Equipment, Consumables), Country (U.S., Canada, Mexico, Germany, Italy, U.K., France, Spain, Netherlands, Belgium, Switzerland, Turkey, Russia, Rest of Europe, Japan, China, India, South Korea, Australia, Singapore, Malaysia, Thailand, Indonesia, Philippines, Rest of Asia- Pacific, Brazil, Argentina, Rest of South America, South Africa, Saudi Arabia, UAE, Egypt, Israel, Rest of Middle East & Africa) Industry Trends and Forecast to 2028

Genetic testing market is expected to gain market growth in the forecast period of 2021 to 2028. Data Bridge Market Research analyses the market to reach at an estimated value of 585.81 billion and grow at a CAGR of 11.85% in the above-mentioned forecast period. Increase in incidences of genetic disorders and cancer drives the genetic testing market.

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The major players covered in the genetic testing market report are 23andMe, Inc., Abbott., Ambry Genetics., BGI, Biocartis, BIO-HELIX, bioMrieux SA, Blueprint Genetics Oy, Cepheid., deCODE genetics, GeneDx, Inc., Exact Sciences Corp, HTG Molecular Diagnostics, Genomictree., Illumina, Inc, Invitae Corporation, Laboratory Corporation of America Holdings, Luminex Corporation., ICON plc, Myriad Genetics, Inc, Natera, Inc., Pacific Biosciences of California, Inc, Pathway Genomics, QIAGEN, Quest Diagnostics Incorporated, F. Hoffmann-La Roche Ltd and Siemens Healthcare Private Limited among other domestic and global players.

Competitive Landscape and Genetic Testing Market Share Analysis

Genetic testing market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, global presence, production sites and facilities, production capacities, company strengths and weaknesses, product launch, product width and breadth, application dominance. The above data points provided are only related to the companies focus related to genetic testing market.

Genetic tests are the type of tests which are defined as medical devices available in the form of kits and panels that are used for testing genetic diseases in humans. The testing is generally performed by collecting samples ofbloodfrom patients and the samples are then run on laboratory machines using test kits. There are numerous types of tests which are used in testing of genetic disorders which includes, predictive and presymptomatic testing, carrier testing, prenatal and newborn testing, diagnostic testing, pharmacogenomic testing among others.

Rise in awareness and acceptance of personalized medicines is the vital factor escalating the market growth, also rising advancements in genetic testing techniques, rising demand for direct-to-consumer genetic testing, rising consumer interest in personalized medicines in Europe, rising application of genetic testing in oncology and genetic diseases in North America and rising physician adoption of genetic tests into clinical care are the major factors among others driving the genetic testing market. Moreover, rising untapped emerging markets in developing countries and rising research and development activities in the machinery used inhealthcarewill further create new opportunities for genetic testing market in the forecasted period of 2021-2028.

However, rising standardization concerns of genetic testing-based diagnostics and rising stringent regulatory requirements for product approvals are the major factors among others which will obstruct the market growth, and will further challenge the growth ofgenetic testing marketin the forecast period mentioned above.

This genetic testing market report provides details of new recent developments, trade regulations, import export analysis, production analysis, value chain optimization, market share, impact of domestic and localised market players, analyses opportunities in terms of emerging revenue pockets, changes in market regulations, strategic market growth analysis, market size, category market growths, application niches and dominance, product approvals, product launches, geographic expansions, technological innovations in the market. To gain more info on genetic testing market contact Data Bridge Market Research for anAnalyst Brief,our team will help you take an informed market decision to achieve market growth.

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Genetic Testing Market Scope and Market Size

Genetic testing market is segmented on the basis of type, technology, application, disease and product. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

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Global Genetic Testing MarketCountry Level Analysis

Genetic testing market is analysed and market size insights and trends are provided by country, type, technology, application, disease and product as referenced above.

The countries covered in the genetic testing market report are U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.

North America dominates the genetic testing market due to rising demand for direct-to-consumer genetic testing and rising consumer interest in personalized medicines. Asia-Pacific is the expected region in terms of growth in genetic testing market due to rise in affordability, increasing surge in healthcare expenditure, and increase in awareness toward early screening of genetic disorders in this region.

The country section of the genetic testing market report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as consumption volumes, production sites and volumes, import export analysis, price trend analysis, cost of raw materials, down-stream and upstream value chain analysis are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of domestic tariffs and trade routes are considered while providing forecast analysis of the country data.

Healthcare Infrastructure growth Installed base and New Technology Penetration

Genetic testing market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipments, installed base of different kind of products for genetic testing market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the genetic testing market. The data is available for historic period 2010 to 2019.

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Global Genetic Testing Market Top Countries Analysis and Manufacturers With Impact of COVID-19 | 2021-2028 Detail Analysis focusing on Application,...

Quebec Premier FranoisLegault, announced the appointment of 43 (35 from 2020) individuals to the National Order of Quebec today, including Dr. Morag Park, Director of McGill Universitys Rosalind and Morris Goodman Cancer Research Centre (GCRC) who was appointed Chevalire. Dr. Park was nominated by Maryse Lassonde, President of the Quebec Superior Council of Education with the support of the Quebec Breast Cancer Foundation (QBCF), with the additional support of McGills Faculty of Medicine and Health Sciences as well as the GCRC.

McGill University is proud that the Government of Quebec has chosen to honour Dr. Morag Park for her outstanding contributions in cancer research, said Martha Crago, Vice-Principal, Research and Innovation. Her work has improved our understanding of cancer and has made a mark on Quebecs culture of research excellence. I extend my heartfeltcongratulationsto Dr. Park on behalf of McGills research community.

It is a great honour to be appointed to the Order of Quebec. I am grateful for my incredible colleagues and trainees in the health and research community who have supported me throughout the years and whose collaborations have enabled the progress weve made to date. However, there remains a long road ahead in the fight against cancer, and support for this research is critical, perhapseven more so as we exit the COVID-19 pandemic, where, like so many fields, cancer care and research has been impacted, said Dr. Morag Park on receiving her appointment.

From the very beginning of her career as a researcher, the Foundation believed in Dr. Morag Parks research projects by awarding her very first research grant in breast cancer. She is a pioneer with her work on breast cancer in Quebec. We are proud to support her since her beginnings and the recognition she receives from the Order of Quebec, said Karine-Iseult Ippersiel, President and CEO of the Quebec Breast Cancer Foundation.

A trailblazer, builder and innovator during a career spanning over thirty years, Dr. Park has made contributions to a wide spectrum of cancer research, from the molecular level to the complex cellular interactions within tissues that dictate the biology of human cancers.

Her trajectory began with her identification and characterization of a key oncogene, the receptor tyrosine kinase (RTK) MET, as a post-doctoral fellow. On this foundation, Dr. Park built a research program of sustained excellence, developing elegant molecular and cell biology approaches to systematically discover key signaling proteins and pathways dictating the functional output of MET and other RTKs which are now key targets in precision medicine for many cancers. Her innovative approach, which set an example followed by many others in the field, identified critical molecular mechanisms of aberrant RTK activation that drive many prevalent cancers. These discoveries have significantly increased our understanding of many crucial processes underlying cancer development, including cell growth and proliferation, survival, motility and invasion and key cell fate decisions.

For the past 20 years, Dr. Park has also built world-leading translational research programs, including innovative pre-clinical models and comprehensive patient sample and data repositories, particularly for breast cancer. These are built on the premise of multidisciplinary collaborations between surgeons, oncologists, pathologists, and informaticians, as well as basic and translational researchers, through her role as Co-Principal Investigator of a Quebec-wide cancer biobanking network of clinicians and researchers. Her work led to a new understanding of the role of non-tumor cells, referred to as the tumor microenvironment in breast cancer, a concept that is now accepted for many cancers. More recently, living biobanks of live cells and tissues harvested during surgeries, developed under Dr. Parks leadership, have allowed the development of patient derived models of breast and other cancers to understand why some patients do not respond to therapy and to develop new therapeutic strategies.

Dr. Parks leadership and expertise in bridging the laboratory and clinic have allowed her to assume leading roles in consortia dedicated to improving outcomes for cancer patients by expanding the reach of precision medicine. These include Qubec Cancer Consortium (QCC), uniting six leading Montreal hospitals, cancer research centres, non-profit and pharmaceutical industry partners, and the Quebec node of the Terry Fox Research Institutes Marathon of Hope Cancer Centres Network (MoHCCN).

Dr. Park has received many prizes and honours including the Canadian Cancer Society (CCS) Robert L. Noble Prize (2017), the Canadian Society for Molecular Biosciences (CSMB) Arthur Wynne Gold Medal Award (2016), and the Grand Prix Scientifique of the Quebec Breast Cancer Foundation (2019). A fellow of the Royal Society of Canada (2007) and of the Canadian Academy of Health Sciences (2017), she was elected Chair of the American Association for Cancer Research (AACR) Tumor Microenvironment Network (2015-2017). She has been recognized consistently through academic appointments of the highest level, including a Distinguished James McGill Professorship (2020-present) and the Diane and Sal Guerrera Chair in Molecular Genetics (2003-present). Nationally, as the Scientific Director of CIHRs Institute of Cancer Research (2008-13), Dr. Park spearheaded key initiatives on personalized medicine, childhood cancers initiation and progression, the role of lifestyle and the environment. She also co-chaired the Canadian Cancer Research Alliance (CCRA), where she led the development of the first Pan-Canadian Cancer Research Strategic Plan. For these and her other efforts she received the CCRA Award for Exceptional Leadership in Cancer Research in 2015.

Congratulations Dr. Park!

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Morag Park named to the Order of Quebec - McGill Reporter - McGill Reporter

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Third Rock Ventures has closed a Series A financing round thatraised $82 million, funding which will be used to launchFlare Therapeutics, a new biotech startup focused on developing precision treatments for cancer and other diseases. Significant contributions were made to the Series A by Boxer Capital, Nextech Invest, Casdin Capital, Invus Financial Advisors, and Eventide Asset Management.

Flare uses a switch-site based drug discovery approach, which helped the company develop a pipeline of therapeutic programs that target pivotal cancer drivers such as transcription factor dysregulation and mutations. The new financing from the Series A will support its ability to advance its lead precision oncology program toward the clinic.

Flare currently has three lead precision oncology programs. The first program is expected to move into the clinic in 2023 or 2024, while the following two programs are expected to enter the clinic in the two years following.

Abbie Celniker, Ph.D., Flares interim Chief Executive Officer, and Third Rock Ventures partner, said in a statement that the new startup company was created to pursue the mission of conquering transcription factors which have been one of the most sought-after targets of drug developers based on the central role they play in cancer and other diseases.

Transcription factors appear to play a central role in cancer, among other diseases, according to emerging data and scientific discoveries published over the past ten years. Yet, transcription factors have continued to be elusive for finding targetable sites for drug discovery, with less than 1% of transcription factors successfully targeted for medicines, according to a statement made by Flares Scientific Co-Founder, Fraydoon Rastinejad, Ph.D., a professor of biochemistry and structural biology at the University of Oxford.

The novel drug discovery paradigm established by Flare uses an approach that targets transcription factors. According to the company, this strategy is based primarily on a broader understanding of the cooperative communication and allosteric interaction among the elements of the transcriptional molecular complex, in contrast to the previous scientific focus on only single, isolated transcription factor domains.

Recent work by the companys Scientific Co-Founders has helped Flare gain a greater understanding of the molecular mechanisms involved in targeting transcription factors. This understanding, according to Flare, has helped its research team to recognize the broad potential to generalize these principles to the switch site as a focal point for drugging transcription factors in a new way.

Scientific Co-Founder of Flare, Steven McKnight, Ph.D., a professor at the University of Texas, Southwestern, noted, Maturation of the fields of human genetics and the biochemistry of gene regulation now point us towards opportunities for therapeutic intervention using conventional, small molecule drugs.

Likely, Flare will also plan to use the new Series A funding to grow its staff to 25 employees. Currently, the company employs 12 to 15 full-time staffers, mainly relying on contractors and partners to fulfill its operational needs.

Original post:
Third Rock Ventures Launches Flare Therapeutics With $82 Million Series A - BioSpace

Over 60 outstanding scientists from all over the globe have joined the Royal Society as Fellows and Foreign Members. The distinguished group of scientists consists of 52 Fellows, 10 Foreign Members and one Honorary Fellow and were all selected for their exceptional contributions to science.

The Royal Society is a self-governing Fellowship made up of the most eminent scientists, engineers and technologists from the UK and the Commonwealth. Its Foreign Members are drawn from the rest of the world.

The Societys fundamental purpose is to recognise, promote, and support excellence in science and to encourage the development and use of science for the benefit of humanity.

The global pandemic has demonstrated the continuing importance of scientific thinking and collaboration across borders, said President of the Royal Society, Sir Adrian Smith.

Each Fellow and Foreign Member bring their area of scientific expertise to the Royal Society and when combined, this expertise supports the use of science for the benefit of humanity.

Our new Fellows and Foreign Members are all at the forefronts of their fields from molecular genetics and cancer research to tropical open ecosystems and radar technology. It is an absolute pleasure and honour to have them join us.

Read the full story and see the list of Cambridge Fellows

Reproduced courtesy of the University of Cambridge

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The Royal Society announces election of new Fellows 2021 - Cambridge Network

UCLA life scientists have identified six "words" that specific immune cells use to call up immune defense genes -- an important step toward understanding the language the body uses to marshal responses to threats.

In addition, they discovered that the incorrect use of two of these words can activate the wrong genes, resulting in the autoimmune disease known as Sjgren's syndrome. The research, conducted in mice, is published this week in the peer-reviewed journal Immunity (Cell Press).

"Cells have evolved an immune response code, or language," said senior author Alexander Hoffmann, the Thomas M. Asher Professor of Microbiology and director of the Institute for Quantitative and Computational Biosciences at UCLA. "We have identified some words in that language, and we know these words are important because of what happens when they are misused. Now we need to understand the meaning of the words, and we are making rapid progress. It's as exciting as when archaeologists discovered the Rosetta stone and could begin to read Egyptian hieroglyphs."

Immune cells in the body constantly assess their environment and coordinate their defense functions by using words -- or signaling codons, in scientific parlance -- to tell the cell's nucleus which genes to turn on in response to invaders like pathogenic bacteria and viruses. Each signaling codon consists of several successive actions of a DNA binding protein that, when combined, elicit the proper gene activation, in much the same way that successive electrical signals through a telephone wire combine to produce the words of a conversation.

The researchers focused on words used by macrophages, specialized immune cells that rid the body of potentially harmful particles, bacteria and dead cells. Using advanced microscopy techniques, they "listened" to macrophages in healthy mice and identified six specific codon-words that correlated to immune threats. They then did the same with macrophages from mice that contained a mutation akin to Sjgren's syndrome in humans to determine whether this disease results from the defective use of these words.

"Indeed, we found defects in the use of two of these words," Hoffmann said. "It's as if instead of saying, 'Respond to attacker down the street,' the cells are incorrectly saying, 'Respond to attacker in the house.'"

The findings, the researchers say, suggest that Sjgren's doesn't result from chronic inflammation, as long thought, but from a codon-word confusion that leads to inappropriate gene activation, causing the body to attack itself. The next step will be to find ways of correcting the confused word choices.

Many diseases are related to miscommunication in cells, but this study, the scientists say, is the first to recognize that immune cells employ a language, to identify words in that language and to demonstrate what can happen when word choice goes awry. Hoffman hopes the team's discovery will serve as a guide to the discovery of words related to other diseases.

"The macrophage is capable of responding to different types of pathogens and mounting different kinds of defenses. The defense units -- army, navy, air force, special operations -- are mediated by groups of genes," he said. "For each immune threat, the right groups of genes must be mobilized. That requires precise and reliable communication with those units about the nature of the threat. NFB dynamics provide the communication code. We identified the words in this code, but we don't yet fully understand how each defense unit interprets the various combinations of the codon-words."

And of course, calling up the wrong unit is not only ineffective, Hoffmann notes, but may do damage, as vehicles destroy roads, accidents happen and worse, as in the case of Sjogren's and, possibly, other diseases.

Then, using an algorithm originally developed in the 1940s for the telecommunications industry, they monitored which of the potential words tended to show up when macrophages responded to a stimulus, such as a pathogen-derived substance. They discovered that six specific dynamical features, or "words," were most frequently correlated with that response.

An analogy would be listening to someone in a conversation and finding that certain three-letter words tend to be used, such as "the," "boy," "toy," and "get," but not "biy" or "bey," said lead author Adewunmi Adelaja, who earned his Ph.D. in Hoffmann's laboratory and is now working toward his M.D. at UCLA.

The team then used a machine learning algorithm to model the immune response of macrophages. If they taught a computer the six words, they asked, would it be able to recognize the stimulus when computerized versions of cells were "talking?" They confirmed that it could. Drilling down further, they explored what would happen if the computer only had five words available. They found that the computer made more mistakes in recognizing the stimulus, leading the team to conclude that all six words are required for reliable cellular communication.

The scientists also used calculus to study the biochemical molecular interactions inside the immune cells that produce the words.

Hoffmann and his colleagues revealed in the journal Science in 2014 how and why the immune system's B cells respond only to true threats. In a study published in Cell in 2013, his team showed for the first time that it was possible to correct a cellular miscommunication involving the connection of receptors to genes during inflammation without severe side effects.

Reference:Adelaja A, Taylor B, Sheu KM, et al. Six distinct NFB signaling codons convey discrete information to distinguish stimuli and enable appropriate macrophage responses. Immunity. 2021;54(5):916-930.e7. doi:10.1016/j.immuni.2021.04.011

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Researchers Decode the "Language" of Immune Cells - Technology Networks

Early identification of unique cell markers is expected to have valuable impact on patents, clinical efficacy, and manufacturing optimization

VANCOUVER, BC / ACCESSWIRE / May 11, 2021 / RepliCel Life Sciences Inc. (OTC PINK:REPCF)(TSXV:RP)(FRA:P6P2) ("RepliCel" or the "Company") is pleased to announce it has now signed and launched a new collaborative research project agreement with the University of British Columbia ("UBC") that enables the second stage of its cell marker research. The project is being co-led by RepliCel's Dr. Kevin McElwee and UBC's Professor Youwen Zhou.

The project commenced in 2017, is designed to deliver a gene and protein expression "map" of healthy hair follicle cells expected to be critically important to improving key components of the manufacturing, regulatory, and clinical profile of RepliCel's cell therapy products.

The initial stage of the study examined multiple cell types from human hair follicles to identify differences, and similarities, in gene and protein expression with the goal of finding markers of interest that would allow potential optimization in target cell isolation from the initial patient tissue biopsy as well as post-manufacturing final product formulation and correlation of specific cellular sub-populations to clinical efficacy. New patents filed around the discoveries made during this collaboration will accrete value to RepliCel's overall technology and product portfolio.

This second stage of the project specifically aims to confirm the presence and in situ localization of cells expressing unique markers selected from the initial project's indicative data and establish robust selection technologies and protocols for the use of the markers in cell isolation and manufacturing of RepliCel's proprietary tissue regenerative cell therapy products.

About Professor Youwen Zhou, M.D., Ph.D.

Dr. Youwen Zhou is a physician-scientist who is a Professor at UBC's Department of Dermatology and Skin Science. He received his BS degree from Nankai University, China, a PhD in Molecular Genetics from the State University of New York, and an MD degree from the University of Toronto. After completing dermatology specialty training at UBC, he joined UBC Faculty of Medicine as a physician-scientist in dermatology in 2000, and was promoted to full professor in 2013. He founded the UBC Molecular Medicine Lab and Chieng Genomics Center at Vancouver Coastal Health Research Institute (VCHRI) with infrastructure funding from the Canada Foundation for Innovation in 2001. Dr. Zhou's research is centered on biomarkers of skin diseases such as skin lymphoma, melanoma, and vitiligo, using a wide variety of methods and approaches, including genome-wide association studies (GWAS), linkage analysis, next generational sequencing, transcriptional profiling, cellular models, as well as genome editing.

Dr. Zhou has published more than 100 articles in journals such as Nature, Cell, Nature Genetics, and Blood, and holds multiple patents in skin lymphoma diagnostic biomarkers. In 2013, Dr. Zhou was awarded a Barney Usher Award for Outstanding Achievements in Dermatology Research from the Canadian Dermatology Association. Dr. Zhou specializes in the diagnosis and treatment of skin cancers and skin pigmentation disorders and is a consultant dermatologist at Vancouver General Hospital and British Columbia Cancer Agency. He teaches graduate students, medical students, dermatology residents, and postdoctoral fellows. Dr. Zhou is the past president of the Canadian Society of Investigative Dermatology and served as a board member for Canadian Institutes for Health Research (CIHR) Institute of Musculoskeletal Health and Arthritis (IMHA). He is also a grant reviewer for CIHR, the Canadian Dermatology Foundation, and the Natural Sciences Foundation of China.

About Dr. Kevin McElwee, Ph.D.

Dr. McElwee, co-discoverer of the Company's technology and co-founder of RepliCel, is a former Professor of Biomedical Sciences at the University of Bradford's Center for Skin Sciences. Previously, he was an Associate Professor in the Department of Dermatology and Skin Health at the University of British Columbia, and Director of the Hair Research Laboratory in the Vancouver Coastal Health Research Institute at Vancouver General Hospital. His research has been funded by competitive grants awarded by multiple organizations including the Canadian Institutes for Health Research (the equivalent of the National Institutes for Health in the USA).

Dr. McElwee is one of only a small group of research scientists worldwide who studies hair biology and associated diseases. He has worked as a hair research scientist for 25 years and has published over 120 medical journal articles and academic book chapters on hair loss research. Dr. McElwee received his Bachelor of Science degree from the University of Aberdeen, Scotland, and his PhD from the University of Dundee, Scotland. Postdoctoral training included three years at the Jackson Laboratory in Maine and four years at the University of Marburg, Germany, studying various hair loss diseases. Dr. McElwee continues to serve as the Company's Chief Scientific Officer.

About RepliCel Life Sciences

RepliCel is a regenerative medicine company focused on developing cell therapies for aesthetic and orthopedic conditions affecting what the Company believes is approximately one in three people in industrialized nations, including aging/sun-damaged skin, pattern baldness, and chronic tendon degeneration. These conditions, often associated with aging, are caused by a deficit of healthy cells required for normal tissue healing and function. These cell therapy product candidates are based on RepliCel's innovative technology, utilizing cell populations isolated from a patient's healthy hair follicles.

The Company's product pipeline is comprised of RCT-01 for tendon repair, RCS-01 for skin rejuvenation, and RCH-01 for hair restoration. RCH-01 is exclusively licensed in Asia to Shiseido Company. RepliCel maintains the rights to RCH-01 for the rest of the world. RCT-01 and RCS-01 are exclusively licensed in Greater China to YOFOTO (China) Health Company. RepliCel and YOFOTO are currently co-developing these products in China. RepliCel maintains the rights to these products outside of Greater China.

RepliCel has also developed a proprietary injection device, RCI-02, and related consumables, which is expected to improve the administration of its cell therapy products and certain other injectables. YOFOTO has exclusively licensed the commercial rights for the RCI-02 device and consumables in Greater China for dermatology applications and is expected to first launch the product in Hong Kong upon it being approved for market launch in either the United States or Europe.

Please visit http://www.replicel.com for additional information.

Notable Facts:

For more information, please contact: Lee Buckler, CEO and President info@replicel.com

SOURCE: RepliCel Life Sciences, Inc.

View source version on accesswire.com: https://www.accesswire.com/645993/RepliCel-Launches-the-Next-Stage-of-a-Research-Project-with-the-University-of-British-Columbia-to-Build-World-Class-Hair-Follicle-Cell-Data-Map

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RepliCel Launches the Next Stage of a Research Project with the University of British Columbia to Build World-Class Hair Follicle Cell Data Map -...

Sperm are simple cells with a straightforward job: swim until they reach an egg, then fertilize it. But in mice, some sperm resort to divisive tactics in order to gain the advantage.

A study published on February 4 in the journal PLOS Genetics shows that a genetic variation in mouse sperm, called the t-type, can give a swimmer the upper hand. These t-type sperm are able to spread a protein called RAC1 that essentially poisons other sperm. T-type sperm plant the seeds of destruction early in their development, then fortify themselves against RAC-1, Brandon Specktor reports for Live Science. When it comes time to race for the egg, the t-type sperm can swim in a straight line while poisoned sperm swim in hapless circles until they die.

We found out that the level of this protein can be more or less active, depending on whether the sperm have the gene to make it, and whether that gene is flipped on like a light switch, says biologist Alexandra Amaral of the Max Planck Institute for Molecular Genetics to Kassidy Vavra at Inverse. The level of protein that is on has to be quite well regulated. If it is too much, sperm don't move well. And if its too low, it also doesnt move well theyre kind of in circles.

T-type sperm produce the RAC1 protein at full throttle.

If all of the sperm in a group are t-type, and theyre all making RAC1, they will all struggle because there is so much of the poisonous protein going around, Sara Rigby reports for Science Focus magazine. On the other hand, if there are no t-type sperm present, then all the other sperm remain relatively healthy and swim well because theres no overabundance of RAC1. However, if a cohort has a mix of t-type and normal sperm, then t-type will have the advantage.

"The trick is that the t-haplotype 'poisons' all sperm, but at the same time produces an antidote, which acts only in t-sperm and protects them," says Bernhard Herrmann, director of the Max Planck Institute for Molecular Genetics, in a statement. "Imagine a marathon, in which all participants get poisoned drinking water, but some runners also take an antidote."

The t-type sperm do the equivalent of poisoning the drinking water early in sperm development, affecting both themselves and their non-variant peers. All of the sperm inherit genes that make it difficult to interpret the chemical signals around them. But in the final cell division of sperm development, when half of a cells genes go to one sperm and the other half to another, only the sperm that inherit the t-type variation have an extra set of genes that reverses the poisons effect, per Live Science.

The poisoned sperm end up swimming in circles, unable to advance in their quest. But the impervious t-type sperm swim ahead. In this case, theres a 99 percent chance that the sperm that fertilizes the egg first will have the t-type variation. The research shows the importance of small genetic variations in sperms success, Amaral tells Inverse.

The study was conducted in about 100 mouse sperm cells, but not all species sperm behave the same way, University of California, Berkeley, cell biologist Polina Lishko tells Inverse. The study is preliminary, but future research could illuminate the specific molecular mechanism behind RAC1 that makes it damaging to sperm at high levels.

An earlier study showed a similar effect of RAC1 on bull sperm, which is more similar to human sperm than a mouses is. Amaral says that the team plans to conduct future research with human sperm, to see if RAC1 might be involved with some cases of male infertility.

Original post:
Mice Sperm Sabotage Other Swimmers With Poison | Smart News - Smithsonian Magazine

Newswise Considered the most lethal form of DNA damage, double-strand breaks must be repaired to prevent cell death. In developing therapies for hard-to-treat breast and ovarian cancers in patients with BRCA gene mutations, scientists aim to identify ways to keep cancer cells from using DNA break repair pathways. New findings demonstrate a previously-unknown capability for polymerase theta (pol theta) a key enzyme in this repair function that shows promise as a new avenue for treatment development.

The study results are published in Molecular Cell.

Researchers at the University of Vermont (UVM), The University of Texas MD Anderson Cancer Center (MD Anderson), and Yale University discovered that pol theta, previously known to extend DNA in the repair process, is also able to behave like a nuclease and trim DNA.

Because these cancer cells rely on the pol theta pathway to survive and repair double-strand breaks, researchers have been focused on pol theta and trying to find out how to inhibit this pathway.

Pol theta is a hot enzyme right now, says senior author and self-described polymerase geek Sylvie Doubli, Ph.D., professor of microbiology and molecular genetics at the UVM Larner College of Medicine and the UVM Cancer Center. This is a new activity for pol theta; its an elegant way of solving the problem you only need one enzyme.

For patients with hard-to-treat cancers, this finding could lead to the development of new therapeutic options, like the Poly-ADP-ribose polymerase (PARP) inhibitors class of drugs that have been used to treat breast and ovarian cancer over the past decade.

The cell has to decide which function needs to be applied and this trimming activity is a point of vulnerability for pol theta, says Doubli. One aim of the research is to create conditions where one reaction can be encouraged over the other.

A potential role for such an inhibitor would be to improve ionizing radiation therapy in cancer patients with BRCA1 or BRCA2 mutations.

Doublis former doctoral student Karl Zahn, Ph.D., now a postdoctoral fellow at Yale, saw evidence of this dual function in pol theta several years ago while working in Doublis lab. He carried out the experiments described in the paper after engaging the expertise of Richard Wood, Ph.D., professor of epigenetics and molecular carcinogenesis at MD Anderson. Wood and Doubli have had a long-term collaboration, funded by a Program Project grant from the National Cancer Institute.

Conducting the experiments, controls, and reproducing the findings took the research team several years but was critical to confirming this discovery.

It was an unexpected finding, and the biochemistry makes sense, suggesting a way to inhibit the DNA repair process orchestrated by pol theta, says Wood.

The trimming reaction is rapid, and many people missed it, says Doubli, adding that the research teams patience and work paid off. Chance favors only the prepared mind, she says, quoting the late French scientist Louis Pasteur.

Excerpt from:
Study Identifies Never-Before-Seen Dual Function in Enzyme Critical for Cancer Growth - Newswise