CRISPR/Cas基因组靶向编辑技术综述

Review about CRISPR/Cas system as a new targeted genome editing technology

Clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated(Cas)是细菌特有的一种获得性免疫系统,研究人员将其改造成为靶向基因组编辑的工具,由于操作简单、成功率高且效率高,成为了靶向基因组编辑工具中的佼佼者。同时CRISPR/Cas系统也被改造作为一种极为有效的位点特异性的转录抑制/激活的工具而在研究工作中广泛应用,不仅如此,具有靶向细胞转录过程中产生的RNA底物的利用前景,从而起到与RNAi技术相类似的效果。CRISPR-Cas技术已经在基因功能研究、动物模型建立、基因治疗等领域得到广泛的推广和应用,有力地推动了相关领域的研究进展。

CRISPR （clustered, regularly interspaced, short, palindromic repeats）/Cas （CRISPR-associated） systems are a unique prokaryotic defense against foreign genetic elements. It also be considered as a targeted genome editing tool in molecular biology research. Because of its simplicity, high success rate and high efficiency in genome targeting, Cas system became the best genome targeting tools compared with ZFNs （Zinc-finger nucleases） and TALENs （Transcrip-tion activator-like effector nucleases）. According to the recent researches, Cas system could be used as an efficient system for site-specific transcriptional repression or activation. Additionally, a specific Cas9 protein has been observed to target an RNA substrate, suggesting that Cas9 may have the same ability as RNAi technology to be programmed to target RNA as well. Overall, the targeted genome editing technology via CRISPR/Cas system has been Widely promot-ed and applied in many field such as the research on gene functions, the the disease model of gene knock-out animal construction and the gene therapy. So it will have significant impact on future advancements in genome engineering.