中国医药导报
中國醫藥導報
중국의약도보
CHINA MEDICAL HERALD
2014年
22期
154-156,164
,共4页
基因靶向修饰技术%Cas9%CRISPR/Cas%基因组编辑%基因治疗
基因靶嚮脩飾技術%Cas9%CRISPR/Cas%基因組編輯%基因治療
기인파향수식기술%Cas9%CRISPR/Cas%기인조편집%기인치료
Gene targeting%Cas9%CRISPR/Cas%Genome editing%Gene therapy
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated(Cas)是细菌特有的一种获得性免疫系统，研究人员将其改造成为靶向基因组编辑的工具，由于操作简单、成功率高且效率高，成为了靶向基因组编辑工具中的佼佼者。同时CRISPR/Cas系统也被改造作为一种极为有效的位点特异性的转录抑制/激活的工具而在研究工作中广泛应用，不仅如此，具有靶向细胞转录过程中产生的RNA底物的利用前景，从而起到与RNAi技术相类似的效果。 CRISPR-Cas技术已经在基因功能研究、动物模型建立、基因治疗等领域得到广泛的推广和应用，有力地推动了相关领域的研究进展。
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated(Cas)是細菌特有的一種穫得性免疫繫統，研究人員將其改造成為靶嚮基因組編輯的工具，由于操作簡單、成功率高且效率高，成為瞭靶嚮基因組編輯工具中的佼佼者。同時CRISPR/Cas繫統也被改造作為一種極為有效的位點特異性的轉錄抑製/激活的工具而在研究工作中廣汎應用，不僅如此，具有靶嚮細胞轉錄過程中產生的RNA底物的利用前景，從而起到與RNAi技術相類似的效果。 CRISPR-Cas技術已經在基因功能研究、動物模型建立、基因治療等領域得到廣汎的推廣和應用，有力地推動瞭相關領域的研究進展。
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated(Cas)시세균특유적일충획득성면역계통，연구인원장기개조성위파향기인조편집적공구，유우조작간단、성공솔고차효솔고，성위료파향기인조편집공구중적교교자。동시CRISPR/Cas계통야피개조작위일충겁위유효적위점특이성적전록억제/격활적공구이재연구공작중엄범응용，불부여차，구유파향세포전록과정중산생적RNA저물적이용전경，종이기도여RNAi기술상유사적효과。 CRISPR-Cas기술이경재기인공능연구、동물모형건립、기인치료등영역득도엄범적추엄화응용，유력지추동료상관영역적연구진전。
CRISPR (clustered, regularly interspaced, short, palindromic repeats)/Cas (CRISPR-associated) systems are a unique prokaryotic defense against foreign genetic elements. It also be considered as a targeted genome editing tool in molecular biology research. Because of its simplicity, high success rate and high efficiency in genome targeting, Cas system became the best genome targeting tools compared with ZFNs (Zinc-finger nucleases) and TALENs (Transcrip-tion activator-like effector nucleases). According to the recent researches, Cas system could be used as an efficient system for site-specific transcriptional repression or activation. Additionally, a specific Cas9 protein has been observed to target an RNA substrate, suggesting that Cas9 may have the same ability as RNAi technology to be programmed to target RNA as well. Overall, the targeted genome editing technology via CRISPR/Cas system has been Widely promot-ed and applied in many field such as the research on gene functions, the the disease model of gene knock-out animal construction and the gene therapy. So it will have significant impact on future advancements in genome engineering.
