CRISPR/Cas9系统在人类疾病中的研究应用进展

Advances in research and application of CRISPR/Cas9 system in human diseases

规律成簇的短回文重复序列(clustered regularly intespaced short palindromic repeats,CRISPR)/(CRISPR associated 9,Cas9)基因编辑技术是由细菌中的获得性免疫系统发展而来的,作为目前最有效最受欢迎的基因编辑工具,Cas9蛋白可在向导RNA的引导下对DNA进行定点切割,与转录激活因子效应物核酸酶(transcription activator-like effector nucleases,TALEN)和锌指核酸酶(Zinc finger nuclease,ZFN)相比效率更高且成本更低。CRISPR/Cas9编辑系统应用于人类疾病研究,有望解决生命科学研究领域多种复杂的分子生物学难题,例如癌症。本文主要从人类疾病的基因治疗出发,重点介绍了该技术在各系统中疾病模型的建立以及在遗传疾病的治疗、病毒感染性疾病治疗、癌症研究等方面的应用,并讨论了这种先进分子技术的未来前景和尚存在的不足。

Clustered regularly intespaced short palindromic repeats/CRISPR associated 9（CRISPR/Cas9）is developed as an adaptive immunity mechanism in prokaryotes. As the most effective and popular geneediting tool,Cas9 protein splice specific DNA sites by binding with short guide RNA molecules,which is better than transcription activator-like effect or nucleases（TALEN）and Zinc finger nuclease（ZFN）. CRISPR/Cas9 system applys for human disease researches and to solve several complex molecular biology problems faced in the life science researches such as cancer. In this review,the recent advancements of CRISPR/Cas9 in the application of disease model establishment will be reviewed,as well as in the protection for hereditary disease,infectious disease,cancer. The future prospect of this advanced molecular technology and the shortcomings have also been discussed.