Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
Jean Bennett(Children's Hospital of Philadelphia), Albert M. Maguire(Children's Hospital of Philadelphia), Francesca Simonelli(University of Naples Federico II), Jennifer Wellman(Children's Hospital of Philadelphia), Jeannette Bennicelli(Penn Presbyterian Medical Center), Bart P. Leroy(Ghent University Hospital), Julie DiStefano‐Pappas(Westat (United States)), Sarah McCague(Children's Hospital of Philadelphia), Eric A. Pierce(Broad Institute), Federico Mingozzi(Inserm), Laura Cyckowski(Children's Hospital of Philadelphia), Junwei Sun(Singapore Management University), Katherine A. High(Spark Therapeutics (United States)), Dominique Cross(Children's Hospital of Philadelphia), Manzar Ashtari(California University of Pennsylvania), J. Fraser Wright(Oxford BioMedica (United Kingdom)), Jason Ruggiero(Penn Presbyterian Medical Center), Daniel C. Chung(Penn Presbyterian Medical Center), Kathleen Marshall(Children's Hospital of Philadelphia), Alberto Auricchio(Federico II University Hospital), Okan U. Elci(Westat (United States)), Puya Aravand(Penn Presbyterian Medical Center)
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