人工核酸酶介导的基因组编辑技术

Genome Editing Technologies Based on Engineered Nuclease

传统的基因组编辑技术是基于胚胎干细胞和同源重组实现生物基因组定向改造,但是该技术打靶效率低,严重制约了生命科学以及医学的研究.因此,研究新的基因组编辑技术十分重要.人工核酸酶介导的基因组编辑技术是通过特异性识别靶位点造成DNA双链断裂,引起细胞内源性的修复机制实现靶基因的修饰.与传统的基因组编辑技术相比,人工核酸酶技术打靶效率高,这对于基因功能的研究、构建人类疾病动物模型以及探索新型疾病治疗方案有着重要的意义.人工核酸酶技术有3种类型:锌指核酸酶(ZFN)、类转录激活因子核酸酶(TALEN)及规律成簇的间隔短回文重复序列(CRISPR).本文将对以上3种人工核酸酶技术的原理以及在生命科学和医学研究的应用进行综述.

Traditional genome editing technologies can be used to modify biological genome by the embryonic stem cells and homologous recombination. However,this technology has low efficiency of gene targeting,which restricted the life sciences and medical research. Therefore,the research of new genome editing technology is very important. Engineered nuclease mediated genome editing technologies create DNA double-strand breaks by specific recognition of target sites,causing intracellular endogenous repair mechanisms to complete the modification of target gene. Compared with traditional genome editing technologies,the engineered nuclease technologies have high efficiency of gene targeting,which has an important significance in the study of gene function,structure of animal models of human disease,and exploration of new disease treatment program. There are three types of the engineered nuclease technologies： ZFN（ zinc finger nucleases）,TALEN（ transcription activator-like effector nucleases） and CRISPR（ clustered regularly interspaced short palindromic repeats）. In this article,we reviewed the application of the engineered nuclease technologies in life sciences and medical research.