CRISPR／Cas靶向编辑技术系统与耳聋动物模型的建立

CRISPR/Cas Targeted Editing Technique System and Animal Model of Deafness

CRISPR(Clustered regularly interspaced short palindromicrepeats)/Cas(CRISPR-associated)系统是新一代的靶向基因组编辑技术系统,其突出特征是Cas蛋白,其通过g RNA(guide RNA)的引导下对外源性的DNA进行靶向切割,从而为细菌入侵核酸提供了免疫力。由CRISPR/Cas系统介导的基因靶向修饰技术成功地应用于多种细菌及多种生物的基因组靶向修饰,对人类基因组DNA的靶向修饰也已经得到了证实,并且CRISPR/Cas系统与以往技术相比在很多方面显示出了的优点。CRISPR/Cas系统在遗传缺陷疾病动物模型的建立技术中已经占有重要地位,而各种类型基因修饰动物模型是目前内耳基因功能、聋病分子机制研究的最理想方法和必备条件。随着耳聋的遗传学研究不断深入,诊断水平的不断提高,对遗传性耳聋动物模型的需求也不断增多,CRISPR/Cas系统介导的基因组靶向修饰技术也会不断完善,该技术将在耳聋的遗传学研究和诊断治疗等多领域发挥更大作用。

The CRISPR （Clustered regularly interspaced short palindromic repeats）/Cas （CRISPR - associated） system is a new generation of targeted genome editing technique system. Its prominent feature is the Cas protein, which is capable of targeted cutting of exogenous DNA under the guidance of gRNA （guide RNA） and provides immunity to bacteria invasion of nu- cleic acids. CRISPR/Cas system, mediated targeted gene modification technology has been successfully has been applied to a variety of bacteria and other biological genomes, as well as to human genomic DNA. Compared previous technology, the CRIS- PR/Cas system has shown a number of advantages on numerous aspects. The CRISPR/Cas system is now an important techni- cal means in establishing animal model of genetic defect diseases. Various types of genetically modified animal models are a preferred approach and essential in studying inner ear gene functions and molecular mechanisms of deafness. Deafness gene studies advance and diagnosis improves, there will be increasing needs for animal models of hereditary deafness. CRISPR/Cas system mediated targeted the genome modification technology will continue to improve and will play an increasingly important role in many fields of genetic research and diagnosis and treatment of deafness.