基因组靶向修饰技术研究进展  

Research progress on the targeted genome modification

作　　者：周阳[1] 袁少飞[1] 蒋廷亚[1] 韩邦兴[2] 高力[1] 陈乃富[2] 

机构地区：[1]江苏大学生命科学研究院,镇江212013 [2]皖西学院生物与制药工程学院中药研究与开发工程技术研究中心,六安237012

出　　处：《生物学杂志》2015年第5期70-75,共6页Journal of Biology

基　　金：国家自然科学基金(31301919);江苏省自然科学基金(BK20130506);安徽省自然科学基金(1508085MH203);安徽省高等学校省级自然科学基金项目(KJ2011A270;KJ2013A265);江苏大学高级人才科研启动基金(1281330018)

摘　　要：基因组靶向修饰技术是研究基因功能的重要方法之一,该技术也被用于人类疾病的治疗上,从而成为近来生物学研究的热点。传统的靶向修饰技术由于其效率低、有毒性等缺点注定其将要被更高效、安全的技术所取代,因此产生了后来的三代基因组靶向修饰技术:锌指核酸酶(Zinc finger nuclease,ZFN)、类转录激活因子效应物核酸酶(Transcription activator-like effector nuclease,TALEN)和常间回文重复序列丛集关联蛋白系统(Clustered regularly interspaced short palindromic repeats/CRISPR-associated proteins 9,CRISPR/Cas9)。这3种技术在克服传统技术缺陷的基础上,也针对其上一代技术的缺陷进行了自身的改善。对三代基因组靶向修饰技术,尤其最近发展起来的CRISPR/Cas9的结构组成、作用原理和基因定点修饰中的应用进行阐述,最后对三代基因组靶向修饰技术进行比较。Targeted genome modification technology is one of the important methods to study genes′function, which also can be used for the treatment of human diseases at the same time.It is becoming a hot field of biology research in recent years.Because of its low efficiency and toxicity, traditional targeted modification technology will be replaced by a more efficient and safer technology.Thus three generations of targeted genome modification technologies have been developed.It includes ZFN, TALEN and CRISPR/Cas9.These three techniques overcome the defects of traditional technology.Here, we discuss the research progress in the structure, mechanism and application of these three technologies, especially newly developed CRISPR/Cas9.Finally, the three generations of genome modifi-cation techniques are compared.

关 键 词：基因组靶向修饰 ZFN TALEN CRISPR/Cas9 

分 类 号：Q78[生物学—分子生物学]