Bart P. Leroy

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

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, Jennifer Wellman, Kathleen Marshall et al. · The Lancet · 2016 · 445 citations · Full text

Translational Medicine, Gene Therapies, Ocular Disease +7

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy

Albert M. Maguire, Stephen R. Russell, Jennifer Wellman et al. · Ophthalmology · 2019 · 362 citations · Full text

Biallelic Mutation of BEST1 Causes a Distinct Retinopathy in Humans

Rosemary Burgess, I. D. Millar, Bart P. Leroy et al. · The American Journal of Human Genetics · 2008 · 307 citations · Full text

Ocular Disease, Ophthalmology, Genetic Disorder +7