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

Albert M. Maguire, 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 IW Morgan, Bernd Hauck, Olga Zelenaia, Xiaosong Zhu, Leslie Raffini, Frauke CoppietersElfride 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*

*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

691 Scopus citations

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Medicine and Dentistry

Biochemistry, Genetics and Molecular Biology