人工核酸内切酶介导的新一代基因组编辑技术进展

Progress of new-generation genome editing mediated by engineered endonucleases

摘要:

对基因组特定位置进行针对性修饰的实验方法称为基因组编辑技术。近年出现的ZFN、TALEN、CRISPR/Cas等一系列人工核酸内切酶逐渐形成了新一代基因组编辑技术，极大地促进了基因组靶向修饰技术的发展，并在基因功能研究、基因治疗等领域开始发挥巨大作用。文中对这一技术的基本原理、发展历程和应用方式进行了简要总结。

Abstract:

Genome editing refers to the experimental methods to targeted modify specific loci in the genomic DNA sequence. In recent years, engineered endonucleases, including ZFN, TALEN and CRISPR/Cas, have been developed as a new-generation genome editing technique, and greatly improved the feasibility of gene function analyses, gene therapy, etc. Here, we briefly summarize the basic principle, developmental process and applications of this technology.