Age-dependent effects of RPE65 gene therapy for Leber’s congenital amaurosis: a phase 1 dose-escalation trial

Por Maguire, Albert M, High, Katherine A, Auricchio, Alberto, Wright, J Fraser, Pierce, Eric A, Testa, Francesco, Mingozzi, Federico, Bennicelli, Jeannette L, Ying, Gui-shuang, Rossi, Settimio, Fulton, Ann, Marshall, Kathleen A, Banfi, Sandro, Chung, Daniel C, Morgan, Jessica IW, Hauck, Bernd, Zelenaia, Olga, Zhu, Xiaosong, Raffini, Leslie, Coppieters, Frauke, De Baere, Elfride, Shindler, Kenneth S, Volpe, Nicholas J, Surace, Enrico M, Acerra, Carmela, Lyubarsky, Arkady, Redmond, T Michael, Stone, Edwin, Sun, Junwei, McDonnell, Jennifer Wellman, Leroy, Bart P, Simonelli, Francesca, Bennett, Jean
Publicado no Lancet (2009)