Tmc gene therapy restores auditory function in deaf mice.
Boston, United States. In Sci Transl Med, Aug 2015
Exogenous Tmc1 or its closely related ortholog, Tmc2, were capable of restoring sensory transduction, auditory brainstem responses, and acoustic startle reflexes in otherwise deaf mice, suggesting that gene augmentation with Tmc1 or Tmc2 is well suited for further development as a strategy for restoration of auditory function in deaf patients who carry TMC1 mutations.
Gene therapy for deafness: How close are we?
Göttingen, Germany. In Sci Transl Med, Aug 2015
Virus-mediated transfer of genes encoding the mechanotransducer channel candidates TMC1 and TMC2 into hair cells of the ear partially restores hearing in animal models of human genetic deafness (Askew et al., this issue).