首页> 中文期刊> 《医学分子生物学杂志》 >CRISPR-Cas9技术在人类疾病治疗中的潜在应用

CRISPR-Cas9技术在人类疾病治疗中的潜在应用

         

摘要

CRISPR-Cas9的发明为生物学领域带来一场新的技术革命。 CRISPR-Cas系统是一种存在于多种细菌和古菌内针对外源遗传物质侵袭的获得性免疫系统,由规律性重复短回文序列簇及其相关蛋白组成。近年该系统被发展成一种由CRISPR相关RNA介导的Cas9核酸内切酶靶向基因编辑技术,在科研、医学及生物技术等多个领域上被广泛应用。 CRISPR-Cas9技术多功能及可编程特点,令其在动物模型制备、物种改良等方面取得重要应用。同时在遗传性疾病、恶性肿瘤、感染性疾病等许多疾病的研究、治疗及预防上均显示出巨大应用潜力。%A new revolution has been sparked in biological field due to the invention of CRISPR-Cas9 technology. CRISPR-Cas system, which is comprised of clustered regularly-inter-spaced short palindromic repeats (CRISPR) and associated proteins (Cas), confers adaptive im-munity against exogenic elements in many bacteria and most archaea. During recent years, this sys-tem has been developed to a CRISPR-associated RNA-guided endonuclease Cas9 targeted genome editing tool. It has been widely applied in many fields such as basic biology, biotechnology and medicine. With the versatile functions and programmability, CRISPR-Cas9 has been greatly used for animal model generation, species improvement and so on. Also it shows great application poten-tial in the study, treatment, and prevention of many human diseases ( including genetic diseases, cancers, infectious diseases, etc) .

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