Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F
Abstract 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...
Main Authors: | , , , , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Portfolio
2023-04-01
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Series: | Nature Communications |
Online Access: | https://doi.org/10.1038/s41467-023-38038-y |