Xiaosong Zhu

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Albert M. Maguire, Francesca Simonelli, Eric A. Pierce et al. · New England Journal of Medicine · 2008 · 2.1K citations · Full text

Ocular Disease, Genetics, Immunology +18

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Stephen R. Russell, Jean Bennett, Jennifer Wellman et al. · The Lancet · 2017 · 1.8K citations · Full text

Ocular Disease, Retina, Ophthalmology +6

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 et al. · The Lancet · 2009 · 849 citations

Gene Therapies, Genetic Disorder, Genetics +7

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

Francesca Simonelli, Albert M. Maguire, Francesco Testa et al. · Molecular Therapy · 2009 · 544 citations · Full text

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

Jean Bennett, Manzar Ashtari, Jennifer Wellman et al. · Science Translational Medicine · 2012 · 387 citations · Full text

Gene Therapies, Translational Medicine, Rpe65 Gene +13