Article
Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success.
Proceedings of the National Academy of Sciences of the United States of America - 26 Apr 2005
Jacobson Samuel G, Aleman Tomas S, Cideciyan Artur V, Sumaroka Alexander, Schwartz Sharon B, Windsor Elizabeth A M, Traboulsi Elias I, Heon Elise, Pittler Steven J, Milam Ann H, Maguire Albert M, Palczewski Krzysztof, Stone Edwin M, Bennett Jean
Abstract excerpt
Mutations in RPE65, a gene essential to normal operation of the visual (retinoid) cycle, cause the childhood blindness known as Leber congenital amaurosis (LCA). Retinal gene therapy restores vision to blind canine and murine models of LCA. Gene therapy in blind humans with LCA from RPE65 mutations may also have potential for success but only if the retinal photoreceptor layer is intact, as in the early-disease...
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