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Gene Therapy Rescues Cone Function In Congenital Achromatopsia, Andras Komaromy, John J. Alexander, Jessica S. Rowlan, Monique M. Garcia, Vincent Chiodo, Asli Kaya, Jacqueline C. Tanaka, Gregory M. Acland, William W. Hauswirth, Gustavo D. Aguirre
Gene Therapy Rescues Cone Function In Congenital Achromatopsia, Andras Komaromy, John J. Alexander, Jessica S. Rowlan, Monique M. Garcia, Vincent Chiodo, Asli Kaya, Jacqueline C. Tanaka, Gregory M. Acland, William W. Hauswirth, Gustavo D. Aguirre
Gustavo D. Aguirre, VMD, PhD
The successful restoration of visual function with recombinant adeno-associated virus (rAAV)-mediated gene replacement therapy in animals and humans with an inherited disease of the retinal pigment epithelium has ushered in a new era of retinal therapeutics. For many retinal disorders, however, targeting of therapeutic vectors to mutant rods and/or cones will be required. In this study, the primary cone photoreceptor disorder achromatopsia served as the ideal translational model to develop gene therapy directed to cone photoreceptors. We demonstrate that rAAV-mediated gene replacement therapy with different forms of the human red cone opsin promoter led to the restoration of cone function …