Safety and Durability of Effect of Contralateral-Eye Administration Of
HHS Public Access Author manuscript Author ManuscriptAuthor Manuscript Author Lancet. Manuscript Author Author manuscript; Manuscript Author available in PMC 2017 March 15. Published in final edited form as: Lancet. 2016 August 13; 388(10045): 661–672. doi:10.1016/S0140-6736(16)30371-3. 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 trial Prof Jean Bennett, MD, Jennifer Wellman, MSc, Kathleen A Marshall, COT, Sarah McCague, BA, Manzar Ashtari, PhD, Julie DiStefano-Pappas, BA, Okan U Elci, PhD, Daniel C Chung, DO, Junwei Sun, MSc, J Fraser Wright, PhD, Dominique R Cross, MPH, Puya Aravand, BA, Laura L Cyckowski, BA, Jeannette L Bennicelli, PhD, Federico Mingozzi, PhD, Alberto Auricchio, MD, Eric A Pierce, MD, Jason Ruggiero, MD, Prof Bart P Leroy, MD, Francesca Simonelli, MD, Katherine A High, MD, and Prof Albert M Maguire, MD Center for Advanced Retinal and Ocular Therapeutics (Prof J Bennett MD, M Ashtari PhD, D C Chung DO, J Sun MSc, P Aravand BA, J L Bennicelli PhD, Prof A M Maguire MD) and F M Kirby Center for Molecular Ophthalmology (Prof J Bennett, M Ashtari, D C Chung, J L Bennicelli, E A Pierce MD, Prof A M Maguire), Department of Ophthalmology (E A Pierce, J Ruggiero MD, Prof A M Maguire), Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, USA; Center for Cellular and Molecular Therapeutics (Prof J Bennett, J Wellman MSc, K A Marshall COT, S McCague BA, J Sun, J F Wright PhD, D R
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