CRISPR/Cas9系统的脱靶效应

doi:10.13560/j.cnki.biotech.bull.1985.2016.03.006

摘要/Abstract

摘要： 在细菌中发现的免疫系统CRISPR/Cas9, 已经成为最有效的基因工程编辑工具, 甚至大有希望可以治疗人类遗传性疾病。但是CRISPR/Cas9系统在使用时会产生严重的脱靶问题, 导致假表型和错误的解释。提高与靶点结合的高效率, 同时减少脱靶效应, 将是今后CRISPR/Cas9技术的挑战。综述关注与CRISPR/Cas9脱靶效应相关的内容, 总结了影响其靶点专一性的因素, 减少CRISPR/Cas9脱靶效应的可行性方法和设计工具等, 供大家学习讨论。

关键词: 脱靶, 靶点专一性, CRISPR/Cas9

Abstract: As an adaptive immune system discovered in bacteria, clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9(Cas9)technology has been used as the most effective genome-editing tool. By now, CRISPR/Cas9 has shown a great promise in healing human genetic diseases, however, off-target is a critical issue while applying it. The off-target of CRISPR/Cas9 leads to the false phenotype and wrong interpretation. Optimization of on-target activity and minimizing off-target activity will be challenges while CRISPR/Cas9 is applied in the future. In this review, we focused on the off-target related issues, including factors involved in the target specificity, strategies and tools of minimizing the off-target of CRISPR/Cas9.

Key words: off-target, target specificity, CRISPR/Cas9

尹珅,贺桂芳,赖方秾,谢凤云,马俊宇. CRISPR/Cas9系统的脱靶效应[J]. 生物技术通报, 2016, 32(3): 31-37.

YIN Shen, HE Gui-fang, LAI Fang-nong, XIE Feng-yun, MA Jun-yu. Off-target of CRISPR/Cas9 System[J]. Biotechnology Bulletin, 2016, 32(3): 31-37.

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