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