Restoration of Hearing in the VGLUT3 Knockout Mouse Using Virally Mediated Gene Therapy

  title={Restoration of Hearing in the VGLUT3 Knockout Mouse Using Virally Mediated Gene Therapy},
  author={Omar Akil and Rebecca P Seal and Kevin Burke and Chuansong Wang and Aurash S Alemi and Matthew John During and Robert H Edwards and Lawrence R Lustig},
Mice lacking the vesicular glutamate transporter-3 (VGLUT3) are congenitally deaf due to loss of glutamate release at the inner hair cell afferent synapse. Cochlear delivery of VGLUT3 using adeno-associated virus type 1 (AAV1) leads to transgene expression in only inner hair cells (IHCs), despite broader viral uptake. Within 2 weeks of AAV1-VGLUT3 delivery, auditory brainstem response (ABR) thresholds normalize, along with partial rescue of the startle response. Lastly, we demonstrate partial… CONTINUE READING
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Regulation of POU4F3 gene expression in hair cells by 50 DNA in mice

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