CRISPR/Cas9基因编辑技术与神经系统疾病的基础研究及临床应用进展

Progress in Basic Research and Clinical Application of CRISPR/Cas9 Gene Editing Technology and Neurological Diseases

规律间隔成簇短回文重复序列及其相关蛋白9(CRISPR/Cas9)系统是一种由获得性免疫系统在微生物中开发的基因组编辑技术,它根据互补碱基配对原理在特定位点插入或移除基因,或修复致病基因突变,具有高效、简便、低廉的特点,在基础研究和疾病治疗领域显示出巨大的潜力。近年来,CRISPR/Cas9基因编辑技术发展迅速,在多种神经疾病模型的构建、致病基因筛选和靶向治疗等方面具有极大的应用潜力,被评估为神经相关类型疾病研究和治疗的一种有前途的方法。未来,对CRISPR/Cas9基因编辑技术进行深入研究有望为神经系统相关疾病提供有效的治疗方法。

The clustered regularly interspaced short palindromic repeats(CRISPR)and CRISPR-associated protein 9(CRISPR/Cas9)system is a genome editing technology developed by the acquired immune system in microorganisms.It inserts or removes genes at specific sites according to the principle of complementary base pairing,or repairs disease-causing gene mutations.It is inexpensive and shows great potential in the fields of basic research and disease treatment.In recent years,the CRISPR/Cas9 gene editing technology has developed rapidly and has great application potential in the construction of various neurological disease models,pathogenic gene screening and targeted therapy,and is evaluated as a promising method for the research and treatment of neural related diseases.In the future,in-depth research on CRISPR/Cas9 gene editing technology is expected to provide effective treatment for nervous system related diseases.