基因编辑技术及其在宿主细胞改造中的运用  

Gene Editing Technique and its Application in Host Cell Transformation

作　　者：王辰 赵伟 WANG Chen;ZHAO Wei(Chia-Tai Tianqing Pharmaceutical Research Institute,Nanjing 211100,China)

机构地区：[1]正大天晴药业集团股份有限公司研究院,江苏南京211100

出　　处：《药物生物技术》2021年第2期192-199,共8页Pharmaceutical Biotechnology

摘　　要：近二十年来,基因编辑技术取得了突飞猛进的发展,从早期的锌指核酸酶(Zinc finger nucleases,ZFNs)技术到最近开发出的规律成簇间隔短回文重复序列(Clustered regularly interspaced short palindromic repeats,CRISPR)技术,均能通过与靶向序列特异性结合的DNA结构域及靶向性核酸内切酶,成功实现简便可控的基因组编辑,目前基因编辑技术已成为基因治疗领域研究强有力的研究工具。除此之外,运用基因编辑技术还可以实现高效、精准、成本合理的宿主细胞改造,为治疗性抗体等生物技术药物的发展开辟新的路径。文章主要综述几种常用基因编辑技术:锌指核酸酶(ZFNs)、转录激活因子样效应蛋白核酸酶(Transcription activator-like effector nucleases,TALEN)技术、规律成簇间隔短回文重复序列(CRISPR)技术和重组腺相关病毒技术(Recombinant adeno-associated viruses,r AAV)的原理、优缺点、专利情况及基因编辑技术在宿主细胞改造中的相关应用。In the past twenty years,gene editing technique has made rapid progress. Eithervia the early Zinc finger nucleases( ZFNs) technique or the recently developed Clustered regularly interspaced short palindromic repeats( CRISPR) technique,researchers can successfully achieve simple and controllable genome editing through DNA domain and targeted endonuclease specifically bound to the targeted sequence. At present,gene editing technique has become a powerful research tool in the field of gene therapy. In addition,gene editing technique can also be used to achieve efficient,accurate and cost-effective host cell transformation,and open up a new path for the development of therapeutic antibodies and other biotechnological drugs. This paper reviewed the principle,advantages and disadvantages,patent situation and the application of gene editing technique in host cell transformation of several common gene editing techniques such as Zinc finger nuclease( ZFNs),Transcription activator like effector nuclease( TALEN),Clustered regularly interspaced short palindromic repeats( CRISPR) and Recombinant adeno-associated viruses( r AAV).

关 键 词：基因编辑技术 GS基因敲除 宿主细胞 ZFNs TALEN CRISPR 

分 类 号：Q789[生物学—分子生物学]