Lentiviral Gene Transfer of Rpe65 Rescues Survival and Function of Cones in a Mouse Model of Leber Congenital Amaurosis

BACKGROUND: RPE65 is specifically expressed in the retinal pigment epithelium and is essential for the recycling of 11-cis-retinal, the chromophore of rod and cone opsins. In humans, mutations in RPE65 lead to Leber congenital amaurosis or early-onset retinal dystrophy, a severe form of retinitis pi...

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Detalhes bibliográficos
Main Authors: Bemelmans, Alexis-Pierre, Kostic, Corinne, Crippa, Sylvain V, Hauswirth, William W, Lem, Janis, Munier, Francis L, Seeliger, Mathias W, Wenzel, Andreas, Arsenijevic, Yvan
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2006
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Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC1592340/
https://ncbi.nlm.nih.gov/pubmed/17032058
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pmed.0030347
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