Article
Gene therapy for hereditary hearing loss by SLC26A4 mutations in mice reveals distinct functional roles of pendrin in normal hearing.
Theranostics - 1 Jan 2019
Kim Min-A, Kim Sung Huhn, Ryu Nari, Ma Ji-Hyun, Kim Ye-Ri, Jung Jinsei, Hsu Chuan-Jen, Choi Jae Young, Lee Kyu-Yup, Wangemann Philine, Bok Jinwoong, Kim Un-Kyung
Abstract excerpt
Rationale: Mutations of SLC26A4 that abrogate pendrin, expressed in endolymphatic sac, cochlea and vestibule, are known to cause autosomal recessive sensorineural hearing loss with enlargement of the membranous labyrinth. This is the first study to demonstrate the feasibility of gene therapy for pendrin-related hearing loss. Methods: We used a recombinant viral vector to transfect Slc26a4 cDNA into embryonic day...
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