基因编辑技术是以特异性改变遗传物质靶向序列为目标的技术。近年来,锌指核酸酶(zinc finger nuclease, ZFN)、类转录激活因子效应核酸酶(transcription activator-like effector nuclease, TALEN)、规律成簇的间隔短回文重复(regular clustering of short palindrome repeats, CRISPR)和单碱基编辑(base editing, BE)技术的相继出现,不仅为基因功能研究提供了有力的工具,还为生命医学提供了新的治疗方案。基因编辑技术已经大范围应用于动物细胞模型的构建、药物靶点的筛查和基因功能研究等,在基因治疗领域也展现出广阔的应用前景。本文就基因编辑技术的研究进展及其在基因治疗中的应用进行了概述,并对基因编辑技术的的原理、发展史、优缺点以及在基因治疗中的应用前景和机遇挑战进行了讨论,以期为基因编辑技术的临床转化提供参考。

Gene editing technologies are used to specifically edit the target sequence. With the development of zinc finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), regular clustering of short palindrome repeats (CRISPR) and single base editing (BE) techniques, gene editing technologies not only provide powerful tools for gene functional studies, but also offer new therapeutic strategies in biomedical research. Gene editing has demonstrated broad application prospects in the gene therapy field, as well as in the construction of animal and cell models, drug target screening and gene functional research. In this review, we summarize several typical gene editing technologies, their characteristics and applications in gene therapy and discusses their opportunities and challenges in gene therapy, thereby providing critical insights and references on the clinical application of gene editing technologies.