Usher syndrome type III: cochlear gene therapy preserves hair cell synaptic transmission to the auditory neurons

Scientists from the Institut Pasteur, Inserm, Sorbonne University and the Collège de France, working in close collaboration with scientists from the University of Bordeaux, have recently elucidated the mechanisms of hearing loss associated with mutations in clarin-1, a molecule that plays an essential role in the auditory system. The absence of clarin-1 leads to a progressive hearing impairment, which could be prevented by gene therapy that targets the sensory hair cells in the inner ear. This discovery offers new prospects for the therapeutic treatment of individuals with hearing loss due to hair cell synaptopathies.

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