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...

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Detalles Bibliográficos
Autores principales: Marcovich, Irina, Baer, Nicholas K., Shubina-Oleinik, Olga, Eclov, Rachel, Beard, Clayton W., Holt, Jeffrey R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
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

Internet

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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