《生命科学》 2015, 27(1): 93-98

CRISPR-Cas9技术在干细胞中的应用

梁　丹，吴宇轩，李劲松*

(中国科学院上海生命科学研究院生物化学与细胞生物学研究所，细胞生物学国家重点实验室，上海 200031)

摘　要：摘　要：成簇规律间隔短回文重复序列系统(Clustered regularly interspaced short palindromic repeats， associated RNA guided endonuclease Cas9 (CRISPR-Cas9) 是细菌或古细菌在长期演化过程中形成的抵御外来遗传物质的一种获得性免疫防御机制，其中II 型CRISPR-Cas 系统依赖Cas9 核酸内切酶靶向剪切外源DNA。Cas9内切酶在向导RNA 的指导下靶向性地剪切特定基因位点，已被广泛应用在不同种属的基因编辑研究中。利用CRISPR-Cas9 基因编辑系统的优势，结合现有干细胞研究技术，在小鼠、大鼠甚至灵长类动物的功能基因组研究中，可以大幅提高各种基因修饰动物的获得效率，缩短获得的时间，从而快捷有效地研究基因功能；同时，可以建立包括灵长类疾病模型在内的多种动物疾病模型，促进生物医学的发展，造福人类。

Progress of CRISPR-Cas9 in stem cell research

LIANG Dan， WU Yu-Xuan， LI Jin-Song*

(State Key Laboratory of Cell Biology， Institute of Biochemistry and Cell Biology，
    Shanghai Institutes for Biological Sciences， Chinese Academy of Sciences， Shanghai， 200031， China)

Abstract: Abstract: Clustered regularly interspaced short palindromic repeats associated RNA guided endonuclease Cas9 (CRISPR-Cas9) is a kind of adaptive immune defense system， which protects the bacteria or archaea against foreign genetic material. Type II CRISPR-Cas system relies on Cas9 endonuclease that cuts exogenous DNA. Cas9 endonuclease targets and attacks the specific gene loci under the guidance of single guide RNA (sgRNA). CRISPR-Cas9 genome-editing technology has been widely used in different species. The application of CRISPR-Cas9 technology in stem cell research can greatly promote the generation of various genetically modified animal models from mammalians， such as mice， rats and primates， which can be used to identify the function of different genes at organismal level. Meanwhile， animal disease models can also be established efficiently， which can be applied to development of new treatment strategies.