CAJ | 학술논문

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。
기인조파향수식기술시연구기인공능적중요방법지일，해기술야피용우인류질병적치료상，종이성위근래생물학연구적열점。전통적파향수식기술유우기효솔저、유독성등결점주정기장요피경고효、안전적기술소취대，인차산생료후래적삼대기인조파향수식기술：자지핵산매（ Zinc finger nuclease， ZFN）、류전록격활인자효응물핵산매（ Transcription activator－like effector nuclease， TALEN）화상간회문중복서렬총집관련단백계통（ Clustered regularly interspaced short palindromic repeats／CRISPR－associated proteins 9， CRISPR／Cas9）。저3충기술재극복전통기술결함적기출상，야침대기상일대기술적결함진행료자신적개선。대삼대기인조파향수식기술，우기최근발전기래적CRISPR／Cas9적결구조성、작용원리화기인정점수식중적응용진행천술，최후대삼대기인조파향수식기술진행비교。
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.