Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9

doi:10.1016/j.stem.2013.10.016

	Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9
	Wu, Yuxuan 1; Liang, Dan 1,2; Wang, Yinghua 1,2; Bai, Meizhu1,3 ; Tang, Wei 4; Bao, Shiming 5; Yan, Zhiqiang 5; Li, Dangsheng 6; Li, Jinsong1,3
	2013-12-05
发表期刊	CELL STEM CELL (IF:19.8[JCR-2023],22.6[5-Year])
ISSN	1934-5909
卷号	13 期号:6 页码:659-662
发表状态	已发表
DOI	10.1016/j.stem.2013.10.016
摘要	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 could be rescued by coinjection into zygotes of Cas9 mRNA and a single-guide RNA (sgRNA) targeting the mutant allele. Correction occurred via homology-directed repair (HDR) based on an exogenously supplied oligonucleotide or the endogenous WT allele, with only rare evidence of off-target modifications. The resulting mice were fertile and able to transmit the corrected allele to their progeny. Thus, our study provides proof of principle for use of the CRISPR-Cas9 system to correct genetic disease.
收录类别	SCI
语种	英语
资助项目	Shanghai Municipal Commission for Science and Technology[12JC1409600] ; Shanghai Municipal Commission for Science and Technology[13XD1404000]
WOS研究方向	Cell Biology
WOS类目	Cell & Tissue Engineering ; Cell Biology
WOS记录号	WOS:000329571700009
出版者	CELL PRESS
WOS关键词	ONE-STEP GENERATION ; CAS9 NUCLEASE ; HUMAN-CELLS ; GENOME ; SYSTEM ; MICE ; SPECIFICITY ; MUTATIONS ; ELEGANS
原始文献类型	Article
引用统计	正在获取...
文献类型	期刊论文
条目标识符	https://kms.shanghaitech.edu.cn/handle/2MSLDSTB/2448
专题	生命科学与技术学院生命科学与技术学院_特聘教授组_李劲松组生命科学与技术学院_博士生
通讯作者	Li, Jinsong
作者单位	1.Chinese Acad Sci, Shanghai Key Lab Mol Androl, Grp Epigenet Reprogramming,Shanghai Inst Biol Sci, State Key Lab Cell Biol,Inst Biochem & Cell Biol, Shanghai 200031, Peoples R China 2.Univ Chinese Acad Sci, Beijing 100049, Peoples R China 3.Shanghai Tech Univ, Sch Life Sci & Technol, Shanghai 200031, Peoples R China 4.Chinese Acad Sci, Shanghai Inst Biol Sci, Inst Biochem & Cell Biol, Anim Core Facil, Shanghai 200031, Peoples R China 5.Chinese Acad Sci, Shanghai Inst Biol Sci, Shanghai Lab Anim Ctr, Shanghai 201615, Peoples R China 6.Chinese Acad Sci, Shanghai Inst Biol Sci, Shanghai Informat Ctr Life Sci, Shanghai 200031, Peoples R China
通讯作者单位	生命科学与技术学院
推荐引用方式 GB/T 7714	Wu, Yuxuan,Liang, Dan,Wang, Yinghua,et al. Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9[J]. CELL STEM CELL,2013,13(6):659-662.
APA	Wu, Yuxuan.,Liang, Dan.,Wang, Yinghua.,Bai, Meizhu.,Tang, Wei.,...&Li, Jinsong.(2013).Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9.CELL STEM CELL,13(6),659-662.
MLA	Wu, Yuxuan,et al."Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9".CELL STEM CELL 13.6(2013):659-662.