CRISPR技术的发展及应用研究进展

Research Progresses on the Development and Application of CRISPR Technology

CRISPR作为一种基于RNA的后天免疫防御系统,其间隔序列与噬菌体或质粒序列存在同源性,能利用靶位点特异性的RNA指导Cas蛋白靶向几乎所有生物和细胞中遗传错误的基因。与锌指核酶ZFN和转录激活因子样效应物核酶TALEN的基因编辑技术相比,更廉价高效,所以自2013年首次应用以来,迅速被广大中外研究人员接受并应用于研究,成为当今生命科学领域研究的新热点。鉴于CRISPR对今后的科学研究及现实应用具有重要意义,对CRISPR研究的最新动态及在基因治疗等方面的应用进行综述,并在此基础上讨论了该技术存在的局限性及解决方法,最后对CRISPR的研究前景进行了展望,以期为相关科研人员了解CRISPR提供参考。

CRISPR,an adaptive immune defense system based on RNA,whose space sequence has homologous with the sequences of phage or plasmid,can use RNA that has specificity in target point to guide the Cas proteins to target the genetic error genes in almost allorganisms and cells. This gene editing technique,compared with zinc finger nuclease（ZFN）and transcription activator-like effector nuclease（TALEN）,is cheaper and more efficient. Therefore,since its first application in 2013,CRISPR has been accepted quickly and applied tothe studies by the masses of Chinese and foreign researchers,becoming a new hot spot in today＇s life science research. Regarding the greatsignificance of CRISPR in future scientific studies and practical applications,this article reviews the recent research progress on CRISPRand its applications in gene therapy,and discusses the limitations of this technology and the solutions. Finally,the foreground of CRISPR is prospected for providing useful reference for relevant researchers.