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Article

Visual Acuity after Retinal Gene Therapy for Choroideremia

27 Apr 2016

Abstract excerpt

Two recent clinical reports of retinal gene therapy with adeno-associated virus (AAV) vectors in patients with Leber’s congenital amaurosis showed initial gains in visual function that subsequently declined. We previously reported early improvement in visual acuity in two of six patients who received retinal gene therapy in one eye (the study eye) to treat choroideremia, a disease that is characterized by atrophy...

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PMID: 27120491
DOI: 10.1056/nejmc1509501

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