CRISPR/Cas9系统介导的DNA碱基编辑研究

Research on DNA Base Editing Mediated by CRISPR/Cas9 System

基因组编辑指的是对于基因组特定位点的序列进行定向的修改,该技术的发展对于基因治疗具有重要的意义。自从CRISPR/Cas9系统被成功应用于哺乳动物细胞实现高效的基因组编辑,就凭借其构建简单和高效的优势发展成为应用最为广泛的基因组编辑工具。CRISPR/Cas9系统可以实现高效的基因敲除、基因敲入、基因上调和基因下调等等。此外,CRISPR/Cas9系统被应用于高效DNA碱基修复,对于遗传病治疗具有重要的意义。本文综述了CRISPR/Cas9系统的发展及其在DNA碱基编辑中的应用。

Genome editing refers to the directional modification of the sequence of specific loci in the genome. The developmentofthis technology is of great significance for gene therapy. Since CRISPR/Cas9 system was successfully applied to mammalian cells to achieve efficient genome editing, it has developed into the most widely used genome editing tool due to its advantages of simple construction and high efficiency. CR1SPR/Cas9 system can realize efficient gene knockout, gene typing, gene up-regulation and gene down - regulation. In addition, the CRISPR/Cas9 system has been applied to efficient DNA base repair, which is ofgreat significance for the treatment of hereditary diseases. This paper reviews the development of CRISPR/Cas9 system and its application in DNA base editing.