Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo

Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo

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Choroideremia (CHM) is an X-linked retinal degeneration of photoreceptors, the retinal pigment epithelium (RPE) and choroid caused by loss of function mutations in the CHM/REP1 gene that encodes Rab escort protein 1. As a slowly progressing monogenic retinal degeneration with a clearly identifiable...

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Bibliographic Details
Main Authors: Tolmachova, T, Tolmachov, O, Barnard, A, Silva, D, Lipinski, D, Walker, N, MacLaren, R, Seabra, M
Format: Journal article
Language:English
Published: 2013

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