CRISPR—Cas9基因编辑技术在构建动物疾病模型中的应用  

Application of CRISPR-Cas9 genome editing for constructing animal models of human diseases

作　　者：欧展辉[1] 孙筱放[1] 

机构地区：[1]广东省产科重大疾病重点实验室,广东省高等教育生殖与遗传重点实验室,广州医科大学附属第三医院妇产科研究所,广州510150

出　　处：《中华医学遗传学杂志》2016年第4期559-563,共5页Chinese Journal of Medical Genetics

基　　金：国家自然科学基金-广东联合基金（U1132005）;国家自然科学基金（31171229）;广东省科技厅计划项目（2013851000087,2014A020212354）;广州市科信局协同创新一期项目（201508020258,201400000003-4,201400000004-4）

摘　　要：CRISPR—Cas9基因编辑技术是一种新的基因编辑技术，可以在靶基因位点直接进行基因组编辑。它由一条导向RNA（shortsingleguideRNA，sgRNA）指导Cas9核酸酶识别和切割双链DNA，诱发非同源末端连接或同源重组，从而实现在目的DNA上进行随机的突变、特定DNA片段插入或内源性突变修复等基因编辑。该技术已成功被用于包括人类体细胞在内的多种细胞、植物及动物上进行基因编辑。本文主要对利用CRISPR—Cas9基因编辑技术构建动物疾病模型的应用进行综述。The CRISPR-Cas9 system is a new targeted nuclease for genome editing, which can directly introduce modifications at the targeted genomic locus. The system utilizes a short single guide RNA （sgRNA） to direct the endonuclease Cas9 in the genome. Upon targeting, Cas9 can generate DNA double- strand breaks （DSBs）. As such DSBs are repaired by non-homologous end joining （NHEJ） or homology directed repair （HDR）, therefore facilitates introduction of random or specific mutations, repair of endogenous mutations, or insertion of DNA elements. The system has been successfully used to generate gene targeted cell lines including those of human, animals arid plants. This article reviews recent advances made in this rapidly evolving technique for the generation of animal models for human diseases.

关 键 词：CRISPR—Cas9 基因编辑 非同源末端连接 同源重组 动物模型 

分 类 号：Q78[生物学—分子生物学] R-332[医药卫生]