Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

Although genetic factors contribute to almost half of all deafness cases, treatment options for genetic deafness are limited(1–5). 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:guid...

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Pubblicato in:Nature
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2017
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5784267/
https://ncbi.nlm.nih.gov/pubmed/29258297
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nature25164
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