Gene Transfer with AAV9-PHP.B Rescues Hearing in a Mouse Model of Usher Syndrome 3A and Transduces Hair Cells in a Non-human Primate

Gene Transfer with AAV9-PHP.B Rescues Hearing in a Mouse Model of Usher Syndrome 3A and Transduces Hair Cells in a Non-human Primate

Hereditary hearing loss often results from mutation of genes expressed by cochlear hair cells. Gene addition using AAV vectors has shown some efficacy in mouse models, but clinical application requires two additional advances. First, new AAV capsids must mediate efficient transgene expression in bot...

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Bibliographic Details
Main Authors: Bence György, Elise J. Meijer, Maryna V. Ivanchenko, Kelly Tenneson, Frederick Emond, Killian S. Hanlon, Artur A. Indzhykulian, Adrienn Volak, K. Domenica Karavitaki, Panos I. Tamvakologos, Mark Vezina, Vladimir K. Berezovskii, Richard T. Born, Maureen O’Brien, Jean-François Lafond, Yvan Arsenijevic, Margaret A. Kenna, Casey A. Maguire, David P. Corey
Format: Article
Language:English
Published: Elsevier 2019-06-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050118301153

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http://www.sciencedirect.com/science/article/pii/S2329050118301153

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