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

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Published in Lancet on October 23, 2009

Authors

Albert M Maguire1, Katherine A High, Alberto Auricchio, J Fraser Wright, Eric A Pierce, Francesco Testa, Federico Mingozzi, Jeannette L Bennicelli, Gui-shuang Ying, Settimio Rossi, Ann Fulton, Kathleen A Marshall, Sandro Banfi, Daniel C Chung, Jessica I W Morgan, Bernd Hauck, Olga Zelenaia, Xiaosong Zhu, Leslie Raffini, Frauke Coppieters, Elfride De Baere, Kenneth S Shindler, Nicholas J Volpe, Enrico M Surace, Carmela Acerra, Arkady Lyubarsky, T Michael Redmond, Edwin Stone, Junwei Sun, Jennifer Wellman McDonnell, Bart P Leroy, Francesca Simonelli, Jean Bennett

Author Affiliations

1: F M Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA.

Associated clinical trials:

Safety Study in Subjects With Leber Congenital Amaurosis | NCT00516477

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis | NCT00999609

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 | NCT01208389

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