Gene Therapy Can Restore Hearing in Adults, First-of-Its-Kind Trial Shows : ScienceAlert
Up to three in every 1,000 newborns has hearing loss in one or both ears. While cochlear implants offer remarkable...
therapy
Gene therapy shines light on congenital stationary night blindness for future cures | Journal of Translational Medicine
Masland RH. The fundamental plan of the retina. Nat Neurosci. 2001;4:877–86.Article PubMed CAS Google Scholar Baden T, Euler T. Retinal...
New discovery shows how viral vectors release genes in therapy
Researchers at The University of Osaka have found how structural changes in the VP1 protein of AAV vectors trigger genome...
A Suite of Enhancer AAVs Support Targeted Gene Therapy for Brain Disease
Different populations of cells in the mouse brain, each one targeted with high specificity by one of the many new...
A Pennsylvania baby is first to receive personalized gene therapy
A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for...
World-first CRISPR therapy could ‘transform’ treatment for rare genetic diseases, but key challenges lie ahead
It has been described as a revolutionary technology — and won its inventors a Nobel Prize.CRISPR gene editing, often simply...
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...
