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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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: Wu, Jason, Solanes, Paola, Nist-Lund, Carl, Spataro, Sofia, Shubina-Oleinik, Olga, Marcovich, Irina, Goldberg, Hannah, Schneider, Bernard L., Holt, Jeffrey R.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2021
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Original Article
Online adgang: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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