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Optimized AAV Vectors for TMC1 Gene Therapy in a Humanized Mouse Model of DFNB7/11
Gene therapy for genetic hearing loss is an emerging therapeutic modality for hearing restoration. However, the approach has not yet been translated into clinical application. To further develop inner-ear gene therapy, we engineered a novel mouse model bearing a human mutation in the transmembrane c...
Autores principales: | Marcovich, Irina, Baer, Nicholas K., Shubina-Oleinik, Olga, Eclov, Rachel, Beard, Clayton W., Holt, Jeffrey R. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9313133/ https://www.ncbi.nlm.nih.gov/pubmed/35883470 http://dx.doi.org/10.3390/biom12070914 |
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