Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.

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BACKGROUND: Choroideremia is an X-linked recessive disease that leads to blindness due to mutations in the CHM gene, which encodes the Rab escort protein 1 (REP1). We assessed the effects of retinal gene therapy with an adeno-associated viral (AAV) vector encoding REP1 (AAV.REP1) in patients with th...

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Bibliographic Details
Main Authors: Maclaren, R, Groppe, M, Barnard, A, Cottriall, C, Tolmachova, T, Seymour, L, Clark, K, During, M, Cremers, F, Black, G, Lotery, A, Downes, S, Webster, A, Seabra, M
Format: Journal article
Language:English
Published: 2014

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