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

著者: Jean Bennett, Jennifer Wellman, Kathleen Marshall, Sarah McCague, Manzar Ashtari, Julie DiStefano‐Pappas, Okan U. Elci, Daniel C. Chung, Junwei Sun, J. Fraser Wright, Dominique Cross, Puya Aravand, Laura Cyckowski, Jeannette Bennicelli, Federico Mingozzi, Alberto Auricchio, Eric A. Pierce, Jason Ruggiero, Bart P. Leroy, Francesca Simonelli, Katherine A. High, Albert M. Maguire - The Lancet 2016 被引用: 456