New era of gene therapy treats baby with ultrarare disease
Dr Kiran Musunuru and Dr Rebecca Ahrens-Nicklas with patient KJ. Credit: Children’s Hospital of Philadelphia In a major step forward...
therapy
In a first, congenital deafness in teens and adults treated with new gene therapy
Up to 3 in every 1,000 newborns has hearing loss in one or both ears. While cochlear implants offer remarkable...
Scientists discover new approach to gene therapy
Credit: bioRxiv (2025). DOI: 10.1101/2025.01.13.632719 Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes...
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...
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...
