Research ArticleDEAFNESS

Tmc gene therapy restores auditory function in deaf mice

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Science Translational Medicine  08 Jul 2015:
Vol. 7, Issue 295, pp. 295ra108
DOI: 10.1126/scitranslmed.aab1996

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Because genetics is a major cause of deafness, Askew and colleagues developed a therapeutic approach to replace mutant genes associated with the mechanotransduction machinery of the inner ear. The gene encoding transmembrane channel–like 1, Tmc1, or its ortholog, Tmc2, was packaged in adeno-associated viral vectors and delivered to mice with mutations in Tmc1 or Beethoven—models representative of autosomal recessive and dominant human deafness, respectively. Both vectors were able to transduce inner hair cells of the mouse cochlea, and partially restore hearing, as determined by auditory brainstem responses and startle reflexes. More than 30 TMC1 mutations have been implicated in recessive prelingual deafness, so it is hoped that this gene therapeutic approach will work long-term to maintain hearing recovery, perhaps supplementary existing technologies such as cochlear implants and hearing aids.