基因修饰动物模(geneticallymodifiedanimalmodels)是研究个体发育过程和疾病中特定基因功能的一种重要工具,在研究人类疾病发生机制、病理生理和评估新药物、新治疗方法方面有重要应用。哺乳动物因其与人类的相似性而常被用来进行人类疾病研究和药物筛选。然而,传统构建基因修饰动物模型的同源重组等方法不仅需要培养的哺乳动物胚胎干细胞系,而且费时费力。近年来新出现的CRISPR—Cas9基因编辑系统可以在哺乳动物中实现快速、准确的基因定点修饰。该文将介绍CRISPR.Cas9系统在构建基因修饰哺乳动物模型中的应用。
Genetically modified animal models are important tools for understanding the gene function in development and disease. They have been widely used for modeling human pathophysiology, drug screening, efficacy evaluation and novel therapy development. However, the conventional technologies via homologous re- combination in embryonic stem cell lines are time-consuming and labor-intense to generate genetically modified mammalian animal models. Recently, the emergence of CRISPR-Cas9 genome editing technology provides a much quick, precise, and site-specific method in the modification of mammalian genomes. This review will focus on the applications of CRISPR-Cas9 in the generation of a variety of genetically modified mammals.