CAJ | 학술논문

基因组编辑技术是一种能精确靶向修饰生物基因组，实现对基因定点敲除和外源基因定点整合的技术。新出现的锌指核酸酶（ZFN）、转录激活子样效应因子核酸酶（TALEN）和规律性重复短回文序列簇与 Cas9蛋白（CRISPRs／Cas9）系统3种新型的基因组编辑技术通过特异性结构识别靶位点，核酸酶发挥切割作用对靶位点进行定点编辑。3种新型基因编辑技术因具有高效准确、制作简单、耗时短等特点而在生命科学研究中得到广泛应用。论文对目前三种新型的基因组定点编辑技术的特点、结构原理、构建方法以及在传统生物模型、功能基因筛选、人类遗传病基因治疗等方面中的应用做一综述。
기인조편집기술시일충능정학파향수식생물기인조，실현대기인정점고제화외원기인정점정합적기술。신출현적자지핵산매（ZFN）、전록격활자양효응인자핵산매（TALEN）화규률성중복단회문서렬족여 Cas9단백（CRISPRs／Cas9）계통3충신형적기인조편집기술통과특이성결구식별파위점，핵산매발휘절할작용대파위점진행정점편집。3충신형기인편집기술인구유고효준학、제작간단、모시단등특점이재생명과학연구중득도엄범응용。논문대목전삼충신형적기인조정점편집기술적특점、결구원리、구건방법이급재전통생물모형、공능기인사선、인류유전병기인치료등방면중적응용주일종술。
Genome editing is a new technology which can modifiy the targeted biological genome accurately so as to knock out some genes site-directly and integrate some exogenous genes site-directly.Recently,sev-eral effective tools were developed quickly,including engineered nuclease mediated zinc-finger nucleases (ZFN),transcription activator-like effector nucleases (TALEN)and clustered regularly interspaced short palindromic repeats along with cas9 protein (CRISPRs/Cas9),and they all can site-directly edit genomes by recognizing the target sites according to the specific structure followed by nuclease cleavage.The three no-vel genome editing technologies have been applied broadly in life science because of their advantages of higher efficiency,making easily and timesaving.Here the characteristics,principles,construction methods of the three novel genome editing technologies and their application in conventional biology model,functional genome screening,and gene therapy for human inherited diseases were reviewed.