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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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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: György, Bence, Meijer, Elise J., Ivanchenko, Maryna V., Tenneson, Kelly, Emond, Frederick, Hanlon, Killian S., Indzhykulian, Artur A., Volak, Adrienn, Karavitaki, K. Domenica, Tamvakologos, Panos I., Vezina, Mark, Berezovskii, Vladimir K., Born, Richard T., O’Brien, Maureen, Lafond, Jean-François, Arsenijevic, Yvan, Kenna, Margaret A., Maguire, Casey A., Corey, David P.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6297893/
https://ncbi.nlm.nih.gov/pubmed/30581889
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.11.003
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