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Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence

1 Nov 2020

Abstract excerpt

Abstract: Subretinal gene therapy trials began with the discovery of RPE65 variants and their association with Leber congenital amaurosis. The RPE65 protein is critical for the normal functioning of the visual phototransduction cascade. RPE65 gene knockout animal models were developed and showed similar diseased phenotypes to their human counterparts. Proof of concept studies were carried out in these animal...

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PMID: 33223822
DOI: 10.2147/opth.s231804

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