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...
Disease
Opus Genetics reports 1-month pediatric results in gene therapy for inherited retinal disease
(Image Credit: AdobeStock/Artinun)Opus Genetics announced the 1-month open-label phase 1/2 results from the first pediatric patients treated with its investigational...
Dallas scientists granted $420K to test gene therapy for rare neurological disease
Childhood-onset striatonigral degeneration is a rare genetic disorder that robs children of the ability to walk and speak by age...
Gene therapy shows promise for treating rare muscle disease
A University of Alberta research team has taken the first step toward proving its innovative gene therapy can successfully treat...
Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms
SUMF1 positive cells in tissues following IT and combined IT/IV gene therapy. Credit: Communications Medicine (2025). DOI: 10.1038/s43856-025-00734-9 In two...
Regional Director’s Keynote Address – Leveraging Technological Innovation for Health Promotion and Disease Prevention: Insights from Asia-Pacific
- Distinguished Delegates and Guests- Partners, colleagues and friend A very good morning to you all. I am very pleased to speak...
New gene therapy research offers hope for people with chronic kidney disease
Hiroyuki Nakai, M.D., Ph.D., works with his team in his Department of Molecular and Medical Genetics lab in the OHSU...
New gene therapy research offers hope for people with chronic kidney disease
Hiroyuki Nakai, M.D., Ph.D., works with his team in his Department of Molecular and Medical Genetics lab in the OHSU...
Gene therapy research offers hope for people with chronic kidney disease
A mechanistic model of AAV9 and AAV-KP1-mediated renal transduction. Credit: Nature Communications (2024). DOI: 10.1038/s41467-024-54475-9 Researchers are making strides in...
