基因编辑及其在疾病治疗中的应用研究进展

Progress of research on gene editing toward therapeutic applications

基因编辑是指对基因组进行定点修饰的一项新技术。利用该技术,可以精确地定位到基因组的某一位点上,在这位点上剪断靶标DNA或RNA片段并插入新的基因片段。此过程通过人工的手段模拟基因的自然突变,进而达到修改编辑生物体原有基因组的目的。基因编辑技术自诞生以来,经历五代发展,技术越来越成熟,编辑精度也日益提高,已经越来越广泛地被应用于包括疾病治疗在内的多种生物学领域,成为近年来的研究热点。本文就基因编辑技术及其在疾病治疗中的应用研究进展进行简要综述。

Gene editing refers to a new technology for site-specific modification of the genome.Using this technology,you can accurately locate a certain site in the genome,cut the target DNA or RNA fragment at this site and insert a new gene fragment.This process simulates the natural mutation of genes by artificial means,and then achieves the purpose of modifying and editing the original genome of the organism.Since its birth,gene editing technology has experienced five generations of development,the technology has become more and more mature,and the editing accuracy has also been improved.It has been more and more widely used in various biological fields including disease treatment and has become a research hotspot in recent years.This article briefly reviews the progress of researches on gene editing technology and the therapeutic applications.