Sarah McCague

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 · 1.9K citations

Medicine, Ophthalmology, Vision Science +14

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 · 456 citations · Full text

Medicine, Genetics, Ophthalmology +16

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

Jean Bennett, Manzar Ashtari, Jennifer Wellman et al. · Science Translational Medicine · 2012 · 391 citations · Full text

Medicine, Immunology, Ophthalmology +17

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 · 391 citations · Full text

Medicine, Ophthalmology, Vision Science +16

Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

Kiran Musunuru, Sarah Grandinette, Xiao Wang et al. · New England Journal of Medicine · 2025 · 278 citations · Full text

Life Sciences, Biotechnology, Molecular Genetics +17