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Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence

Subretinal gene therapy trials began with the discovery of RPE65 variants and their association with Leber congenital amaurosis. The RPE65 protein is critical for the normal functioning of the visual phototransduction cascade. RPE65 gene knockout animal models were developed and showed similar disea...

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Detalles Bibliográficos
Autores principales:	Gao, Jie, Hussain, Rehan M, Weng, Christina Y
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Dove 2020
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7671481/ https://www.ncbi.nlm.nih.gov/pubmed/33223822 http://dx.doi.org/10.2147/OPTH.S231804

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