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Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F
Usher syndrome type 1 F (USH1F), caused by mutations in the protocadherin-15 gene (PCDH15), is characterized by congenital deafness, lack of balance, and progressive blindness. In hair cells, the receptor cells of the inner ear, PCDH15 is a component of tip links, fine filaments which pull open mech...
Autores principales: | Ivanchenko, Maryna V., Hathaway, Daniel M., Klein, Alex J., Pan, Bifeng, Strelkova, Olga, De-la-Torre, Pedro, Wu, Xudong, Peters, Cole W., Mulhall, Eric M., Booth, Kevin T., Goldstein, Corey, Brower, Joseph, Sotomayor, Marcos, Indzhykulian, Artur A., Corey, David P. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10133396/ https://www.ncbi.nlm.nih.gov/pubmed/37100771 http://dx.doi.org/10.1038/s41467-023-38038-y |
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