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Article

Clinical gene therapy for the treatment of RPE65-associated Leber congenital amaurosis

8 Feb 2011

Abstract excerpt

INTRODUCTION: The positive results of pioneering clinical trials using gene therapy as treatment for patients with Leber congenital amaurosis (LCA) have ushered in a new era of molecular retinal therapeutics for LCA, other blinding retinal disorders and gene therapy applications. AREAS COVERED: This review describes the role of retinal pigment epithelium-specific 65 kDa protein (RPE65) in the visual cycle and how...

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Identifiers

PMID: 21299439
DOI: 10.1517/14712598.2011.557358

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