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

	Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9
	Wu, YX; Liang, D; Wang, YH; Bai, MZ; Tang, W; Bao, SM; Yan, ZQ; Li, DS; Li, JS
刊名	CELL STEM CELL
	2013
卷号	13 期号:6 页码:659-662
通讯作者	Li, JS (reprint author), 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.,jsli@sibcb.ac.cn
英文摘要	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.
学科主题	Cell Biology
类目[WOS]	Cell & Tissue Engineering ; Cell Biology
关键词[WOS]	ONE-STEP GENERATION ; CAS9 NUCLEASE ; HUMAN-CELLS ; GENOME ; SYSTEM ; MICE ; SPECIFICITY ; MUTATIONS ; ELEGANS
收录类别	SCI
语种	英语
WOS记录号	WOS:000329571700009
内容类型	期刊论文
版本	出版稿
源URL	[http://202.127.25.143/handle/331003/397]
专题	上海生化细胞研究所_上海生科院生化细胞研究所
推荐引用方式 GB/T 7714	Wu, YX,Liang, D,Wang, YH,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, YX.,Liang, D.,Wang, YH.,Bai, MZ.,Tang, W.,...&Li, JS.(2013).Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9.CELL STEM CELL,13(6),659-662.
MLA	Wu, YX,et al."Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9".CELL STEM CELL 13.6(2013):659-662.