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DOI | 10.1126/science.aaq0180 |
RNA editing with CRISPR-Cas13 | |
Cox, David B. T.1,2,3,4,5,6; Gootenberg, Jonathan S.1,2,3,4,7; Abudayyeh, Omar O.1,2,3,4,6; Franklin, Brian1,2,3,4; Kellner, Max J.1,2,3,4; Joung, Julia1,2,3,4; Zhang, Feng1,2,3,4 | |
2017-11-24 | |
发表期刊 | SCIENCE
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ISSN | 0036-8075 |
EISSN | 1095-9203 |
出版年 | 2017 |
卷号 | 358期号:6366页码:1019-1027 |
文章类型 | Article |
语种 | 英语 |
国家 | USA |
英文摘要 | Nucleic acid editing holds promise for treating genetic disease, particularly at the RNA level, where disease-relevant sequences can be rescued to yield functional protein products. Type VI CRISPR-Cas systems contain the programmable single-effector RNA-guided ribonuclease Cas13. We profiled type VI systems in order to engineer a Cas13 ortholog capable of robust knockdown and demonstrated RNA editing by using catalytically inactive Cas13 (dCas13) to direct adenosine-to-inosine deaminase activity by ADAR2 (adenosine deaminase acting on RNA type 2) to transcripts in mammalian cells. This system, referred to as RNA Editing for Programmable A to I Replacement (REPAIR), which has no strict sequence constraints, can be used to edit full-length transcripts containing pathogenic mutations. We further engineered this system to create a high-specificity variant and minimized the system to facilitate viral delivery. REPAIR presents a promising RNA-editing platform with broad applicability for research, therapeutics, and biotechnology. |
领域 | 地球科学 ; 气候变化 ; 资源环境 |
收录类别 | SCI-E |
WOS记录号 | WOS:000416590400033 |
WOS关键词 | GENOME ; GUIDE ; ADAR1 ; IFIH1 ; BASE |
WOS类目 | Multidisciplinary Sciences |
WOS研究方向 | Science & Technology - Other Topics |
URL | 查看原文 |
引用统计 | |
文献类型 | 期刊论文 |
条目标识符 | http://119.78.100.173/C666/handle/2XK7JSWQ/197385 |
专题 | 地球科学 资源环境科学 气候变化 |
作者单位 | 1.Massachusetts Inst Technol MIT & Harvard, Broad Inst, Cambridge, MA 02142 USA; 2.MIT, McGovern Inst Brain Res, 77 Massachusetts Ave, Cambridge, MA 02139 USA; 3.MIT, Dept Brain & Cognit Sci, E25-618, Cambridge, MA 02139 USA; 4.MIT, Dept Biol Engn, 77 Massachusetts Ave, Cambridge, MA 02139 USA; 5.MIT, Dept Biol, 77 Massachusetts Ave, Cambridge, MA 02139 USA; 6.MIT, Harvard Mit Div Hlth Sci & Technol, Cambridge, MA 02139 USA; 7.Harvard Med Sch, Dept Syst Biol, Boston, MA 02115 USA |
推荐引用方式 GB/T 7714 | Cox, David B. T.,Gootenberg, Jonathan S.,Abudayyeh, Omar O.,et al. RNA editing with CRISPR-Cas13[J]. SCIENCE,2017,358(6366):1019-1027. |
APA | Cox, David B. T..,Gootenberg, Jonathan S..,Abudayyeh, Omar O..,Franklin, Brian.,Kellner, Max J..,...&Zhang, Feng.(2017).RNA editing with CRISPR-Cas13.SCIENCE,358(6366),1019-1027. |
MLA | Cox, David B. T.,et al."RNA editing with CRISPR-Cas13".SCIENCE 358.6366(2017):1019-1027. |
条目包含的文件 | 条目无相关文件。 |
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