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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6297893/
https://www.ncbi.nlm.nih.gov/pubmed/30581889
http://dx.doi.org/10.1016/j.omtm.2018.11.003

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6297893/
https://www.ncbi.nlm.nih.gov/pubmed/30581889
http://dx.doi.org/10.1016/j.omtm.2018.11.003

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