Genome editing is the modification of genomic DNA at a specific target site in a wide variety of cell types and organisms, including insertion, deletion and replacement of DNA, resulting in inactivation of target genes, acquisition of novel genetic traits and correction of pathogenic gene mutations. Due to the advantages of simple design, low cost, high efficiency, good repeatability and short-cycle, CRISPR-Cas systems have become the most widely used genome editing technology in molecular biology laboratories all around the world. In this review, an overview of the CRISPR-Cas systems will be introduced, including the innovations, the applications in human disease research and gene therapy, as well as the challenges and opportunities that will be faced in the practical application of CRISPR-Cas systems.

基因组编辑是在多种细胞类型和生物体的特定靶位点对基因组DNA进行修饰，包括DNA的插入、删除和替换，从而导致靶基因失活、获得新的遗传性状以及纠正致病基因突变。由于设计简单、成本低、效率高、重复性好、周期短等优点，CRISPR-Cas系统已成为全球分子生物学实验室应用最广泛的基因组编辑技术。本文将介绍CRISPR-Cas系统的概况，包括CRISPR-Cas系统的创新、在人类疾病研究和基因治疗中的应用，以及CRISPR-Cas系统在实际应用中将面临的挑战和机遇。 。