Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutatons: a follow-on phase 1 trial

BACKGROUND: Safety and efficacy have been shown in a phase 1 dose-escalation study involving a unilateral subretinal injection of a recombinant adeno-associated virus (AAV) vector containing the RPE65 gene (AAV2-hRPE65v2) in individuals with inherited retinal dystrophy caused by RPE65 mutations. Thi...

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Bibliografiske detaljer
Udgivet i:Lancet
Main Authors: Bennett, Jean, Wellman, Jennifer, Marshall, Kathleen A, McCague, Sarah, Ashtari, Manzar, DiStefano-Pappas, Julie, Elci, Okan U, Chung, Daniel C, Sun, Junwei, Wright, J Fraser, Cross, Dominique R, Aravand, Puya, Cyckowski, Laura L, Bennicelli, Jeannette L, Mingozzi, Federico, Auricchio, Alberto, Pierce, Eric A, Ruggiero, Jason, Leroy, Bart P, Simonelli, Francesca, High, Katherine A, Maguire, Albert M
Format: Artigo
Sprog:Inglês
Udgivet: 2016
Fag:
Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5351775/
https://ncbi.nlm.nih.gov/pubmed/27375040
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S0140-6736(16)30371-3
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