Gene therapy for young children with congenital blindness
- PMID: 39986734
- DOI: 10.1016/S0140-6736(25)00232-6
Gene therapy for young children with congenital blindness
Conflict of interest statement
AVC and TSA are supported by US National Eye Institute, US Food and Drug Administration, Foundation for the National Institutes of Health, Foundation Fighting Blindness, Eye on the Future, BCM Families Foundation, Research to Prevent Blindness, Samuel G Jacobson, MD, PhD, Memorial Fund, Atsena Therapeutics, Editas Medicine, and Opus Genetics. AVC has consulted for Thea, Opus Genetics, Alkeus, Ray Therapeutics, VC Cell Therapy, Astellas, and Huigene (none of which are pursuing treatments relevant to AIPL1-related retinal dystrophy). AVC and TSA are named inventors on several patents owned by the University of Pennsylvania; none are related to AIPL1-related retinal dystrophy or the subjects discussed in this Comment.
Comment on
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Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study.Lancet. 2025 Feb 22;405(10479):648-657. doi: 10.1016/S0140-6736(24)02812-5. Lancet. 2025. PMID: 39986747
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