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Article

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2.

Ophthalmology - 1 Jun 2013

Testa Francesco, Maguire Albert M, Rossi Settimio, Pierce Eric A, Melillo Paolo, Marshall Kathleen, Banfi Sandro, Surace Enrico M, Sun Junwei, Acerra Carmela, Wright J Fraser, Wellman Jennifer, High Katherine A, Auricchio Alberto, Bennett Jean, Simonelli Francesca

Abstract excerpt

OBJECTIVE: The aim of this study was to show the clinical data of long-term (3-year) follow-up of 5 patients affected by Leber congenital amaurosis type 2 (LCA2) treated with a single unilateral injection of adeno-associated virus AAV2-hRPE65v2. DESIGN: Clinical trial. PARTICIPANTS: Five LCA2 patients with RPE65 gene mutations. METHODS: After informed consent and confirmation of trial eligibility criteria, the...

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Identifiers

PMID: 23474247
DOI: 10.1016/j.ophtha.2012.11.048
PMCID: PMC3674112

Genes

RPE65

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