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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5784267/
https://www.ncbi.nlm.nih.gov/pubmed/29258297
http://dx.doi.org/10.1038/nature25164

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5784267/
https://www.ncbi.nlm.nih.gov/pubmed/29258297
http://dx.doi.org/10.1038/nature25164

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