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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Publicat a:Mol Ther
Autors principals: Wu, Jason, Solanes, Paola, Nist-Lund, Carl, Spataro, Sofia, Shubina-Oleinik, Olga, Marcovich, Irina, Goldberg, Hannah, Schneider, Bernard L., Holt, Jeffrey R.
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2021
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7934577/
https://ncbi.nlm.nih.gov/pubmed/33212302
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.11.016
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