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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...
Autores principales: | Wu, Jason, Solanes, Paola, Nist-Lund, Carl, Spataro, Sofia, Shubina-Oleinik, Olga, Marcovich, Irina, Goldberg, Hannah, Schneider, Bernard L., Holt, Jeffrey R. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2021
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Materias: | |
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 |
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