Article
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo.
Nature biotechnology - 1 Mar 2016
Yin Hao, Song Chun-Qing, Dorkin Joseph R, Zhu Lihua J, Li Yingxiang, Wu Qiongqiong, Park Angela, Yang Junghoon, Suresh Sneha, Bizhanova Aizhan, Gupta Ankit, Bolukbasi Mehmet F, Walsh Stephen, Bogorad Roman L, Gao Guangping, Weng Zhiping, Dong Yizhou, Koteliansky Victor, Wolfe Scot A, Langer Robert, Xue Wen, Anderson Daniel G
Abstract excerpt
The combination of Cas9, guide RNA and repair template DNA can induce precise gene editing and the correction of genetic diseases in adult mammals. However, clinical implementation of this technology requires safe and effective delivery of all of these components into the nuclei of the target tissue. Here, we combine lipid nanoparticle-mediated delivery of Cas9 mRNA with adeno-associated viruses encoding a sgRNA...
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