精准序列替换基因组编辑技术研究进展

Research Progress of Genome Editing Technologies for Precise Sequence Replacement

利用基因组编辑技术可以对微生物、动植物和人类细胞系基因组进行精准的序列替换,加速生物育种进程和遗传性疾病治疗,从而在农业生产和医疗上取得突破。基因组序列替换策略主要分为2种:第1种依赖DNA双链断裂,包括将CRISPR-Cas分别与同源重组、单链退火、微同源末端连接等DNA修复途径相结合,或由位点特异性重组系统介导,实现精准的序列替换;第2种依赖DNA单链断裂,主要包括引导编辑、碱基编辑器等技术。本研究综述了不同精准序列替换策略和技术及相关研究进展,理清各策略和技术的优缺点,有助于根据基因组编辑的目的,选择适合的技术和方法实现精准高效的序列替换。

Genome editing technologies have been used for precise genome sequence replacement in microbe,animals,plants,and human cell lines and thus speeding up the process of biological breeding and genetic disease treatment,making a breakthrough in agricultural production and medicine.Two kinds of strategies are commonly utilized for precise sequence replacement.The first kind relies on DNA double-strand break to realize precise sequence replacement,including the combination of CRISPR-Cas system with DNA repair pathway such as homologous recombination,single strand annealing and micro-homologous end joining,and site-specific-recombination-mediated precise sequence replacement.The second kind relies on the DNA single-strand break and mainly includes the technical strategy such as prime editing and base editor.This study mainly reviews the progress on different genome editing strategies and related technologies for precise sequence replacement.This will be of help to choosing the most suitable technical strategy according to the advantages and disadvantages of them,to realize precise and efficient sequence replacement.