Gene therapyinherited retinal diseaseretinitis pigmentosagenetic vectorInherited retinal diseases (IRDs) comprise a heterogeneous group of genetic disorders affecting the retina. Caused by mutations in over 300 genes, IRDs result in visual impairment due to dysfunction and degeneration of photoreceptors, ...
Gene Therapy for Inherited Retinal Diseases: From Laboratory Bench to Patient Bedside and Beyond Article Open access 19 December 2023 Focused Update on AAV-Based Gene Therapy Clinical Trials for Inherited Retinal Degeneration Article 02 November 2020 Review of gene therapies for age-related macula...
Inherited retinal diseases are due to mutations in a broad range of genes leading to partial or total vision loss. Learn how genes are delivered for therapy
Gene therapy strategies for the treatment of inherited retinal diseases have made major advances in recent years. This review focuses on adeno-associated viral (AAV) vector approaches to treat retinal degeneration and, thus, prevent or delay the onset of blindness. Data from human clinical trials ...
Inherited retinal diseases meet their therapeutic match Frank Joseph Goes, MD October 22nd 2024 The retina is a key proving ground for long-held theories of genetics. Read More AAO 2024: New gene therapy shows promise for treatment of retinitis pigmentosa ...
retinal areas with macro- and microdetachments. Analysis of the treated animals confirmed that the gene therapy had restored the “zipper”-like connection between the RPE and PR cells. When the eyes of treated dogs were exposed to light, cell separation didn’t occur in areas of t...
Gene therapy for inherited retinal and optic nerve degenerations. Expert. Opin. Biol. Ther. 18, 37–49 (2018). Article CAS PubMed Google Scholar Bainbridge, J. W. et al. Long-term effect of gene therapy on Leber’s congenital amaurosis. N. Engl. J. Med. 372, 1887–1897 (2015)....
Earlier this year, the FDA approved two other gene therapies that re-engineer people's own immune cells to treat blood cancers, rather than genetic diseases. "The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases...
enjoy improved vision for years to come,” said Dr. Comander. “This project has been underway for 20 years, and the early successes with this therapy demonstrated by Jean Bennett, Al Maguire, and others, inspired me to dedicate my career toward helping patients with inherited retinal ...
A form of childhood blindness called RPE65-Leber's congenital amaurosis, LCA, is the first — and to this date the only — inherited retinal disease for which correctivegene therapyis currently being tested in three on-going clinical trials. This treatment was first developed and perfected in ...