Usher syndrome: gene therapy restores hearing and balance

Scientists from the Institut Pasteur, Inserm, the CNRS, Collège de France, University Pierre et Marie Curie, and the University of Clermont Auvergne, have recently restored hearing and balance in a mouse model of Usher syndrome type 1G (USH1G) characterized by profound congenital deafness and vestibular disorders caused by severe dysmorphogenesis of the mechanoelectrical transduction apparatus of the inner ear’s sensory cells. By locally injecting the USH1G gene, critical for the formation and maintenance of the hair bundle, the mechanosensory antenna of these cells, the scientists successfully restored the function and the structure of this apparatus, resulting in the first recovery of hearing and balance by gene therapy for this mouse model. These findings, published in the journal PNAS, open up new possibilities for the development of gene therapy treatments for hereditary forms of deafness.

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