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

door Bennett, Jean, Wellman, Jennifer, Marshall, Kathleen A, McCague, Sarah, Ashtari, Manzar, DiStefano-Pappas, Julie, Elci, Okan U, Chung, Daniel C, Sun, Junwei, Wright, J Fraser, Cross, Dominique R, Aravand, Puya, Cyckowski, Laura L, Bennicelli, Jeannette L, Mingozzi, Federico, Auricchio, Alberto, Pierce, Eric A, Ruggiero, Jason, Leroy, Bart P, Simonelli, Francesca, High, Katherine A, Maguire, Albert M
Gepubliceerd in Lancet (2016)