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Article

Adeno‐associated virus 8‐mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models

1 May 2014

Abstract excerpt

BACKGROUND: Choroideremia (CHM) is a slowly progressive X-linked retinal degeneration that results in a loss of photoreceptors, retinal pigment epithelium and choroid. CHM, the gene implicated in choroideremia, encodes Rab escort protein-1 (REP-1), which is involved in the post-translational activation via prenylation of Rab proteins. METHODS: We evaluated AAV8.CBA.hCHM, a recombinant adeno-associated virus...

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Identifiers

PMID: 24962736
DOI: 10.1002/jgm.2768

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