Single and Dual Vector Gene Therapy with AAV9-PHP.B Rescues Hearing in Tmc1 Mutant Mice

AAV-mediated gene therapy is a promising approach for treating genetic hearing loss. Replacement or editing of the Tmc1 gene, encoding hair cell mechanosensory ion channels, is effective for hearing restoration in mice with some limitations. Efficient rescue of outer hair cell function and lack of h...

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Detalles Bibliográficos
Autores principales: Wu, Jason, Solanes, Paola, Nist-Lund, Carl, Spataro, Sofia, Shubina-Oleinik, Olga, Marcovich, Irina, Goldberg, Hannah, Schneider, Bernard L., Holt, Jeffrey R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7934577/
https://www.ncbi.nlm.nih.gov/pubmed/33212302
http://dx.doi.org/10.1016/j.ymthe.2020.11.016

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7934577/
https://www.ncbi.nlm.nih.gov/pubmed/33212302
http://dx.doi.org/10.1016/j.ymthe.2020.11.016

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