Junwei Sun

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

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

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

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