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Ophthalmology|August 25, 2019
Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 TrialsAlbert M Maguire, Stephen Russell, Jennifer A Wellman, et al.
Human Molecular Genetics|February 21, 2012
Nxnl2 splicing results in dual functions in neuronal cell survival and maintenance of cell integrityCéline Jaillard, Aurélie Mouret, Marie-Laure Niepon, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 23, 2008
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transferJeannette Bennicelli, John Fraser Wright, Andras Komaromy, et al.
Nature Medicine|January 12, 2021
Exome-wide evaluation of rare coding variants using electronic health records identifies new gene-phenotype associationsJoseph Park, Anastasia M Lucas, Xinyuan Zhang, et al.
Science Translational Medicine|February 11, 2012
AAV2 gene therapy readministration in three adults with congenital blindnessJean Bennett, Manzar Ashtari, Jennifer Wellman, et al.
The Journal of Clinical Investigation|May 25, 2011
Disruption of intraflagellar protein transport in photoreceptor cilia causes Leber congenital amaurosis in humans and miceKarsten Boldt, Dorus A Mans, Jungyeon Won, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 3, 2009
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administrationFrancesca Simonelli, Albert M Maguire, Francesco Testa, et al.
Ophthalmology|June 17, 2022
Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy TrialTomas S Aleman, Rachel M Huckfeldt, Leona W Serrano, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 29, 2006
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injectionSamuel G Jacobson, Gregory M Acland, Gustavo D Aguirre, et al.
Lancet (London, England)|July 5, 2016
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 trialJean Bennett, Jennifer Wellman, Kathleen A Marshall, et al.
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