Article
Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents.
Nature - 11 Jan 2018
Gao Xue, Tao Yong, Lamas Veronica, Huang Mingqian, Yeh Wei-Hsi, Pan Bifeng, Hu Yu-Juan, Hu Johnny H, Thompson David B, Shu Yilai, Li Yamin, Wang Hongyang, Yang Shiming, Xu Qiaobing, Polley Daniel B, Liberman M Charles, Kong Wei-Jia, Holt Jeffrey R, Chen Zheng-Yi, Liu David R
Abstract excerpt
Although genetic factors contribute to almost half of all cases of deafness, treatment options for genetic deafness are limited. We developed a genome-editing approach to target a dominantly inherited form of genetic deafness. Here we show that cationic lipid-mediated in vivo delivery of Cas9-guide RNA complexes can ameliorate hearing loss in a mouse model of human genetic deafness. We designed and validated,...
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