Ontology highlight
ABSTRACT:
SUBMITTER: Pan B
PROVIDER: S-EPMC5340578 | biostudies-literature | 2017 Mar
REPOSITORIES: biostudies-literature
Pan Bifeng B Askew Charles C Galvin Alice A Heman-Ackah Selena S Asai Yukako Y Indzhykulian Artur A AA Jodelka Francine M FM Hastings Michelle L ML Lentz Jennifer J JJ Vandenberghe Luk H LH Holt Jeffrey R JR Géléoc Gwenaëlle S GS
Nature biotechnology 20170206 3
Because there are currently no biological treatments for hearing loss, we sought to advance gene therapy approaches to treat genetic deafness. We focused on Usher syndrome, a devastating genetic disorder that causes blindness, balance disorders and profound deafness, and studied a knock-in mouse model, Ush1c c.216G>A, for Usher syndrome type IC (USH1C). As restoration of complex auditory and balance function is likely to require gene delivery systems that target auditory and vestibular sensory c ...[more]