Machine learning model to predict the fitness of AAV capsids for gene therapy
image: The first peer-reviewed journal in the field of human gene therapy, providing all-inclusive coverage of the research, methods, and...
Genetics and Gene Therapy Research
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...
Enhancing precision in cancer treatment: the role of gene therapy and immune modulation in oncology
1 Introduction The concept of gene therapy dates back to the 1960s, but its clinical application became a reality in...
NSW Health & Medical Research
By masking a virus that delivers genes to cells, more people, including children with rare genetic diseases, may be able...
New study reveals the curative potential of g
image: Adeno-associated virus vector containing an adenine base editor provides an effective strategy to treat hereditary hearing loss caused by the...
Opus Genetics Announces Presentations at Association for
Durham, NC., March 05, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene...
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...
UMass Researchers Advance Gene Therapy for MSUD
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The Future of Gene-Editing Treatments for Rare Diseases < Yale School of Medicine
Angelman syndrome is a rare genetic disorder caused by a mutation on chromosome 15, which hinders the production of a...
