CRISPR/Cas系统：RNA靶向的基因组定向编辑新技术
收稿日期: 2013-07-16
修回日期: 2013-09-04
网络出版日期: 2013-10-25
基金资助
国家自然科学基金项目(编号：383601, 31071479, 31200273)和转基因重大专项(编号：2013ZX08002-004, 2013ZX08002-005 和2013ZX08010-002)资助
CRISPR/Cas: a novel way of RNA-guided genome editing
Received date: 2013-07-16
Revised date: 2013-09-04
Online published: 2013-10-25
CRISPR/Cas系统广泛存在于细菌及古生菌中, 是机体长期进化形成的RNA指导的降解入侵病毒或噬菌体DNA的适应性免疫系统。对Ⅱ型CRISPR/Cas系统的改造使其成为继锌指核酸酶(ZFNs)和TALE核酸酶(TALENs)以来的另一种对基因组进行高效定点修饰的新技术, 与ZFNs和TALENs相比, CRISPR/Cas系统更简单, 并且更容易操作。文章重点介绍了Ⅱ型CRISPR/Cas系统的基本结构、作用原理及这一技术在基因组定点修饰中的应用, 剖析了该技术可能存在的问题, 展望了CRISPR/Cas系统的应用前景, 为开展这一领域的研究工作提供参考。
关键词： CRISPR/Cas系统; 基因组定点修饰; 向导RNA
李君 张毅 陈坤玲 单奇伟 王延鹏 梁振 高彩霞 . CRISPR/Cas系统：RNA靶向的基因组定向编辑新技术[J]. 遗传, 2013 , 35(11) : 1265 -1273 . DOI: 10.3724/SP.J.1005.2013.01265
Bacteria and archaea have evolved an adaptive immune system, known as typeⅡprokaryotic clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system, which uses short RNA to direct the degradation of target sequences present in invading viral and plasmid DNAs. Recent advances in CRISPR/Cas system provide an improved method for genome editing, showing robust and specific RNA-guided endonuclease activity at targeted endogenous genomic loci. It is the latest technology to modify genome DNA specifically and effectively following zinc finger nucleases (ZFNs) and TALE nucleases (TALENs). Compared with ZFNs and TALENs, CRISPR/Cas is much simpler and easier to engineer. This review summarizes recent progress, and discusses the prospects of CRISPR/Cas system, with an emphasis on its structure, principle, applications and potential challenges.
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