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52 results for "gene therapy"
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.
Texas A&M researchers have created a breakthrough map showing how cells control immunity and inflammation. This discovery could lead to new treatments for cancer and inflammatory diseases.
A 19-year-old from Kelowna, B.C., became the first Canadian cured of a life-threatening immune disease using groundbreaking gene editing technology. His doctor says he no longer needs to worry about deadly infections sneaking past his weakened immune system.
Scientists can now track what's happening inside a living brain using just a blood test. This breakthrough could revolutionize how we monitor and treat brain diseases.
When two Tennessee mothers learned their children share an ultra-rare neurological disorder, they discovered something remarkable: a cure exists. Now they're hosting a superhero run to help bring that gene therapy to their kids and fewer than 500 other patients worldwide.
The FDA just unveiled a breakthrough framework that could bring life-saving gene therapies to thousands of children with rare diseases in a fraction of the time. For families who've watched their kids suffer while treatments stall, this changes everything.
A 19-year-old from British Columbia became the first person ever cured using "prime editing," a breakthrough gene therapy that fixed a fatal immune disorder. The treatment freed him from daily medication and the constant fear of life-threatening infections.
Nemours Children's Hospital in Wilmington can now offer a groundbreaking one-time gene therapy that could give children with sickle cell disease a chance at a transformed life. The FDA-approved treatment addresses the root genetic cause of the disease that affects 100,000 Americans and shortens life expectancy by more than 20 years.
The FDA just announced a new pathway that could bring life-saving gene editing treatments to patients with rare diseases in record time. For families battling conditions once considered untreatable, this could change everything.
The FDA just unveiled a new approval process that could bring life-saving treatments to millions of Americans with rare diseases in months instead of years. Families who've heard "wait for the science to catch up" finally have reason to hope.
The FDA just made it easier for life-saving gene therapies to reach patients with ultra-rare diseases who previously had few treatment options. The new pathway means families won't have to wait years for cures when the science shows real promise.
The FDA just made it easier for millions of Americans with rare diseases to access life-saving gene therapies without waiting years for traditional clinical trials. Families who were told to "wait for science to catch up" now have real hope.
Scientists used a precise gene-editing tool to fix a rare brain disorder in mice, reversing learning problems and behavioral issues after birth. The breakthrough suggests that genetic brain damage might not be permanent, offering new hope for treating neurodevelopmental conditions.
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.
A 9½-month-old baby became the first person ever to receive a custom gene-editing treatment designed specifically for his unique mutation. The breakthrough offers hope for thousands of rare genetic diseases that were once considered unreachable.
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.
A Tennessee family is turning their son's rare disease diagnosis into a community rally for hope and research funding. The Walk for a Cure event next Saturday will support gene therapy research for PKAN, a terminal neurological condition affecting only one to three people per million worldwide.
Adults with a rare genetic disease called cystinosis no longer need dozens of pills each day after receiving a groundbreaking one-time gene therapy. Five years later, most patients show stable or improved health across multiple organs.
Scientists created the first detailed maps showing how genes control each other in Alzheimer's brains, uncovering hundreds of targets for future treatments. The AI system named SIGNET goes beyond linking genes to revealing which ones actually drive the disease.
Austin Louis spent his entire life in excruciating pain from sickle cell disease until a revolutionary gene therapy treatment made him feel like "a new human." The 20-year-old New Jersey man became the first patient at Children's Hospital of Philadelphia to receive the FDA-approved CRISPR treatment outside clinical trials.
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