Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders

Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders

Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders, Published online: 22 January 2019; doi:10.1038/s41467-018-08264-w

Mutations in the mechanotransduction channel component TMC1/2 cause deafness. Here, the authors use a synthetic AAV to replace TMC1 and 2 in the inner ear and show restoration of cochlear and vestibular function, of neuronal reponses in the auditory cortex and of hearing and balance in mice.

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