AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

Demonstration of safe and stable reversal of blindness after a single unilateral subretinal injection of a recombinant adeno-associated virus (AAV) carrying the RPE65 gene (AAV2-hRPE65v2) prompted us to determine whether it was possible to obtain additional benefit through a second administration of...

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Main Authors: Bennett, Jean, Ashtari, Manzar, Wellman, Jennifer, Marshall, Kathleen A., Cyckowski, Laura L., Chung, Daniel C., McCague, Sarah, Pierce, Eric A., Chen, Yifeng, Bennicelli, Jeannette L., Zhu, Xiaosong, Ying, Gui-shuang, Sun, Junwei, Wright, J. Fraser, Auricchio, Alberto, Simonelli, Francesca, Shindler, Kenneth S., Mingozzi, Federico, High, Katherine A., Maguire, Albert M.
Format: Artigo
Language:Inglês
Published: 2012
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4169122/
https://ncbi.nlm.nih.gov/pubmed/22323828
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3002865
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