‘Curiosity-driven research’ led to a recent major medical breakthrough. But it’s under threat
Earlier this year news broke about doctors in London curing blindness in children with a rare genetic condition. The genetic...
Genetics and Gene Therapy Research
Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment
News ReleaseThursday, May 15, 2025 NIH-supported gene-editing platform lays groundwork to rapidly develop treatments for other rare genetic diseases.A research...
Desperately ill baby healed with personalized gene therapy, doctors say
A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing...
World’s First Patient Treated with Personalized CRISPR Therapy
CHOP’s Rebecca Ahrens-Nicklas, MD, PhD, and Penn Medicine’s Kiran Musunuru, MD, PhD. Credit: CHOP PHILADELPHIA & NEW ORLEANS – In...
Novel Digital PCR Approaches Advance Gene Therapy Research and Production
Gene therapy (introducing genetic material into living cells to fix, replace, enhance, or block a faulty gene) is rapidly gaining...
Apertura Gene Therapy and Rett Syndrome Research Trust
NEW YORK and TRUMBULL, Conn., April 30, 2025 (GLOBE NEWSWIRE) -- Apertura Gene Therapy, a biotechnology company focused on innovative...
Apertura Gene Therapy and Rett Syndrome Research Trust Collaborate to Pioneer Advanced Genetic Medicines for Rett Syndrome Using TfR1-Targeted AAV Capsid
NEW YORK and TRUMBULL, Conn., April 30, 2025 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy...
Gene therapy developed for maple syrup urine disease shows promise, new UMass Chan study reports
A study led by UMass Chan Medical School researchers demonstrated that a gene therapy to correct a mutation that causes...
The hunt for cell and gene therapies for eye diseases
Microscopy image showing the optic nerve in advanced glaucoma disease. Stem-cell therapy could help restore vision loss caused by degenerative...
