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Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9
文献类型：期刊论文
| 作者 | Wu, YX; Liang, D; Wang, YH; Bai, MZ; Tang, W; Bao, SM; Yan, ZQ; Li, DS; Li, JS |
| 刊名 | CELL STEM CELL
 |
| 出版日期 | 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 |
| 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. |
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