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Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success
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...
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主要な著者: | , , , , , , , , , , , , , |
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フォーマット: | Artigo |
言語: | Inglês |
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National Academy of Sciences
2005
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主題: | |
オンライン・アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC1087926/ https://ncbi.nlm.nih.gov/pubmed/15837919 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0500646102 |
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