基于CRISPR-Cas9技术的基因治疗研究进展

Research progress of CRISPR-Cas9 system for gene therapy

摘要:

CRISPR-Cas9系统是细菌在与噬菌体抗争的进化过程中产生的一种抵御外源DNA入侵的机制，能有效识别并剪切外源DNA。基于其识别切除外源DNA的原理，CRISPR-Cas9系统被开发成为新一代基因编辑工具。与ES打靶、ZFN、TALEN等技术途径相比，CRISPR-Cas9系统操作简便、效率高、成本低，有着极其广阔的应用前景。本文整理了近年内有关CRISPR-Cas9系统的最新文献报道，对该系统工作原理以及针对基因治疗的研究进展进行综述。

Abstract:

The clustered regulatory interspaced short palindromic repeat-Cas9 (CRISPR-Cas9) system is the part of the prokaryotic immune system, which could recognize and delete the exogenous sequences originated from virus or plasmid. Based on its mechanism, CRISPR-Cas9 system was developed into the new generation of gene editing tool. Compared to the existed technologies such as ES targeting, ZFN or TALEN, CRISPR-Cas9 system is a more efficient, economical and promising approach to manipulate the genome. In this review, we summarize the research progress about CRISPR-Cas9 technology, especially the latest applications in gene therapy studies of human diseases.