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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。
The CRISPR-Cas9 system is a mechanism that bacteria resist in the evolutionary process of phage to resist invasion of exogenous DNA and can effectively recognize and cut exogenous DNA. Based on the principle of identifying exogenous DNA, the CRISPR-Cas9 system was developed as a new generation of gene editing tools. Compared with ES targeting, ZFN, TALEN and other technical approaches, the CRISPR-Cas9 system has the advantages of simple operation, high efficiency and low cost, and has extremely broad application prospects. In this paper, the latest literature on CRISPR-Cas9 system has been collected in recent years. The working principle of this system and the progress of gene therapy research are summarized.