Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
Albert M. Maguire(Children's Hospital of Philadelphia), Katherine A. High(Children's Hospital of Philadelphia), Alberto Auricchio(Telethon Institute Of Genetics And Medicine), J. Fraser Wright(Children's Hospital of Philadelphia), Eric A. Pierce(Children's Hospital of Philadelphia), Francesco Testa(University of Naples Federico II), Federico Mingozzi(Children's Hospital of Philadelphia), Jeannette Bennicelli(Penn Presbyterian Medical Center), Gui‐Shuang Ying(Penn Presbyterian Medical Center), Settimio Rossi(University of Naples Federico II), Ann Fulton(Boston Children's Hospital), Kathleen Marshall(Children's Hospital of Philadelphia), Sandro Banfi(Telethon Institute Of Genetics And Medicine), Daniel C. Chung(Penn Presbyterian Medical Center), Jessica I. W. Morgan(Penn Presbyterian Medical Center), Bernd Hauck(Children's Hospital of Philadelphia), Olga Zelenaia(Children's Hospital of Philadelphia), Xiaosong Zhu(Children's Hospital of Philadelphia), Leslie Raffini(Children's Hospital of Philadelphia), Frauke Coppieters(Ghent University Hospital), Elfride De Baere(Ghent University Hospital), Kenneth S. Shindler(Penn Presbyterian Medical Center), Nicholas J. Volpe(Penn Presbyterian Medical Center), Enrico Maria Surace(Telethon Institute Of Genetics And Medicine), Carmela Acerra(University of Naples Federico II), Arkady Lyubarsky(Penn Presbyterian Medical Center), T. Michael Redmond(National Institutes of Health), Edwin M. Stone(University of Iowa), Junwei Sun(Children's Hospital of Philadelphia), Jennifer Wellman McDonnell(Children's Hospital of Philadelphia), Bart P. Leroy(Ghent University Hospital), Francesca Simonelli(University of Naples Federico II), Jean Bennett(Children's Hospital of Philadelphia)
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Abstract
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