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Article

AAV-mediated photoreceptor transduction of the pig cone-enriched retina

17 Mar 2011

Abstract excerpt

Recent success in clinical trials supports the use of adeno-associated viral (AAV) vectors for gene therapy of retinal diseases caused by defects in the retinal pigment epithelium (RPE). In contrast, evidence of the efficacy of AAV-mediated gene transfer to retinal photoreceptors, the major site of inherited retinal diseases, is less robust. In addition, although AAV-mediated RPE transduction appears efficient,...

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PMID: 21412286
DOI: 10.1038/gt.2011.3

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