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Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Bennett, Jean, Jennifer Wellman, Kathleen Marshall, Sarah McCague, Manzar Ashtari, Julie DiStefano-Pappas, Okan Elci, et al. 2016. “Safety and Durability of Effect of Contralateral-Eye Administration of AAV2 Gene Therapy in Patients With Childhood-Onset Blindness Caused by RPE65 Mutations: A Follow-on Phase 1 Trial.”. Lancet.