CRISPR-Cas9系统在疾病模型中的应用

CRISPR-Cas9 System and Its Applications in Disease Models

规律成簇间隔短回文重复(CRISPR)及相关核酸内切酶(Cas)系统是最近发现的一种关于RNA指导核酸内切酶的基因编辑技术,这一技术的发现促进了生物学和医学研究的发展。CRISPR-Cas9系统的简便性使其广泛应用于细胞基因组编辑、动物模型的构建及疾病模型的基因治疗。现就CRISPR-Cas9系统的结构特点、作用机制及应用进行了综述。

Clustered regularly interspaced short palindromic repeats（ CRISPR） and their associated proteins（ Cas） is a gene editing technique of which RNA guiding endonuclease to cleavage genome. The discovery of this technology promoted biological and medicinal studies. This simplicity of the CRISPR-Cas9 system has enabled its widespread applications in cell genome editing,animal models generation and gene therapy of disease models. The structure characteristics,mechanism and applications of CRISPR-Cas9 system were focused on.