Article
Mutation-agnostic RNA interference with engineered replacement rescues Tmc1-related hearing loss.
Life science alliance - 1 Mar 2023
Iwasa Yoichiro, Klimara Miles J, Yoshimura Hidekane, Walls William D, Omichi Ryotaro, West Cody A, Shibata Seiji B, Ranum Paul T, Smith Richard Jh
Abstract excerpt
Hearing loss is the most common sensory deficit, of which genetic etiologies are a frequent cause. Dominant and recessive mutations in TMC1, a gene encoding the pore-forming subunit of the hair cell mechanotransduction channel, cause DFNA36 and DFNB7/11, respectively, accounting for ∼2% of genetic hearing loss. Previous work has established the efficacy of mutation-targeted RNAi in treatment of murine models of...
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