Correction of a genetic disease in mouse via use of CRISPR-Cas9

The CRISPR-Cas9 system has been employed to generate mutant alleles in a range of different organisms. However, so far there have not been reports of use of this system for efficient correction of a genetic disease. Here we show that mice with a dominant mutation in Crygc gene that causes cataracts...

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Veröffentlicht in:	Cell stem cell Jg. 13; H. 6; S. 659
Hauptverfasser:	Wu, Yuxuan, Liang, Dan, Wang, Yinghua, Bai, Meizhu, Tang, Wei, Bao, Shiming, Yan, Zhiqiang, Li, Dangsheng, Li, Jinsong
Format:	Journal Article
Sprache:	Englisch
Veröffentlicht:	United States 05.12.2013
Schlagworte:	Animals Base Sequence Cataract - genetics Cataract - therapy Clustered Regularly Interspaced Short Palindromic Repeats - genetics CRISPR-Associated Proteins - metabolism Disease Models, Animal gamma-Crystallins - genetics gamma-Crystallins - therapeutic use Genetic Therapy Mice Molecular Sequence Data RNA, Messenger - genetics RNA, Messenger - metabolism
ISSN:	1875-9777, 1875-9777
Online-Zugang:	Weitere Angaben
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