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Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

Hao Yin,Chun-Qing Song,Joseph R Dorkin,Lihua J Zhu,Yingxiang Li,Qiongqiong Wu, Angela Park,Junghoon Yang,Sneha Suresh,Aizhan Bizhanova,Ankit Gupta,Mehmet F Bolukbasi,Stephen Walsh,Roman L Bogorad,Guangping Gao,Zhiping Weng,Yizhou Dong,Victor Koteliansky,Scot A Wolfe,Robert Langer,Wen Xue,Daniel G Anderson

NATURE BIOTECHNOLOGY（2016）

引用 720|浏览15
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摘要
Cas9-mediated gene editing corrects hereditary tyrosinemia in a mouse model.
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关键词
Targeted gene repair,Nucleic-acid therapeutics
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