Authors: Carl A. Nist-Lund, Bifeng Pan, Amy Patterson, Yukako Asai, Tianwen Chen, Wu Zhou, Hong Zhu, Sandra Romero, Jennifer Resnik, Daniel B. Polley, Gwenaelle S. Géléoc, & Jeffrey R. Holt

Abstract: Fifty percent of inner ear disorders are caused by genetic mutations. To develop treatments for genetic inner ear disorders, we designed gene replacement therapies using synthetic adeno-associated viral vectors to deliver the coding sequence for Transmembrane Channel-Like (Tmc) 1 or 2 into sensory hair cells of mice with hearing and balance deficits due to mutations in ... Read more about New published paper with support from the Bertarelli Program: Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders