Cost Utility of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease

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

Medicine, Genetics, Ophthalmology +17

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

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 · 858 citations

Medicine, Genetics, Molecular Genetics +12

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 · 551 citations · Full text

Medicine, Immunology, Ophthalmology +15

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