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21190 results for "gene therapy research"
After 20 years of groundbreaking brain research, a powerhouse science team is pivoting to develop gene therapies for Alzheimer's and Parkinson's. Their expertise could unlock new treatments for millions living with brain diseases.
A 21-year-old bioengineering student diagnosed with a rare muscle disease at 13 is now engineering a potential cure for herself and nearly 1 million others worldwide. Héloïse Hoffmann just won a $100,000 fellowship to advance her groundbreaking gene therapy research.
Researchers at the University of Virginia and Northwestern University have created tiny particles that could permanently reduce eye pressure in glaucoma patients with just one injection. The breakthrough gene therapy could eliminate the need for daily eye drops and invasive surgery within five years.
Two Philadelphia research centers just received millions in federal funding to develop gene therapies that could cure rare diseases within three years. The ambitious timeline could bring hope to families who've been waiting decades for treatments.
Scientists in Newcastle are testing a groundbreaking gene therapy that could stop nephronophthisis, the top cause of kidney failure in children and young adults. Using kidney cells found in urine samples, researchers are working to fix the genetic glitch that steals healthy childhoods.
Researchers in Tokyo have developed a breakthrough molecule that delivers DNA into cells without causing harmful inflammation, a major obstacle in current gene therapies. Early tests in mice show the new method works up to 14 times better than existing approaches.
After decades of research, gene therapy is bringing lasting relief to sickle cell patients after just one treatment. UCLA researchers are leading the charge to cure a disease that affects 100,000 Americans.
Scientists have begun testing a breakthrough gene therapy that could restore hearing for people with the most common genetic cause of deafness. Skylark Bio just dosed their first patient, marking a major step toward helping millions born with this genetic mutation.
A nonprofit and patient foundation just committed $7.65 million to develop a gene therapy for a rare muscular dystrophy with no current treatments. The partnership will take the therapy from initial design all the way to human clinical trials.
Children born completely deaf are hearing whispers for the first time thanks to an experimental gene therapy that has shown remarkable success in the largest study of its kind. The treatment could become the first FDA-approved gene therapy for deafness within months.
Researchers who developed the first FDA-approved gene therapy for inherited blindness are among this year's $3 million Breakthrough Prize winners. Their work is opening doors for treatments that were once considered impossible.
Swedish researchers have cracked the code on why promising gene therapies get stuck in labs instead of reaching patients. Their new "Navigator" system brings hospitals, universities, and innovators together from day one to fast-track treatments from breakthrough to bedside.
An eight-month-old infant in Israel became the first person to receive groundbreaking gene therapy that restores missing brain function, potentially saving children from a devastating form of epilepsy. The treatment represents years of research turning into real hope for families facing ultra-rare genetic diseases.
When doctors couldn't help her son's rare brain disorder, Amber Freed learned genetics, coordinated researchers worldwide, and raised millions to create the first gene therapy for his condition. Maxwell, now 8, became the first person ever to receive the groundbreaking treatment.
Children as young as 5 can now benefit from groundbreaking CRISPR gene therapy for severe blood disorders. New research shows the treatment works safely in younger kids, potentially preventing years of disease complications.
Scientists at UC San Diego have developed a gene therapy that shields brain cells from toxic proteins linked to dementia, successfully preserving memory in mice. The treatment could offer hope for millions facing Alzheimer's, frontotemporal dementia, and ALS.
Philadelphia researchers just won the prestigious 2026 Breakthrough Prize for developing a gene therapy that restores vision in children born with a rare form of blindness. The treatment has already helped hundreds of patients see for the first time.
Three major research institutions joined forces to create a nonprofit that will develop affordable gene therapies for rare diseases and share their methods with doctors worldwide. The initiative could transform personalized treatments from million-dollar fundraising campaigns into standard medical procedures.
Researchers at USC are using AI to fast-track lifesaving cell and gene therapies for children with rare diseases, transforming how treatments reach the smallest patient populations. The new framework could help families access cutting-edge cures months or even years sooner.
Scientists at University of Rochester developed a gene therapy that uses the brain's natural cleaning system to deliver treatments throughout the brain, avoiding one of medicine's toughest obstacles. The breakthrough could transform treatment for multiple sclerosis, Huntington's disease, and rare childhood brain disorders.
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