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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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書誌詳細
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: National Academy of Sciences 2005
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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