CRISPR/Cas9技术在基因治疗中的应用及其优化方案初探

Exploration on the application of CRISPR/Cas9 technology in gene therapy and optimization scheme

基因编辑是指对DNA核苷酸序列进行定点修饰的一项技术,该技术可精确剪断靶向DNA片段并插入新的基因片段,是基因工程中的一项重要技术。规律成簇的间隔短回文重复(CRISPR)技术的出现,标志着基因编辑技术进入了新的领域,为基因功能的研究和疾病的治疗提供了极大的帮助。本文对CRISPR系统的原理和在基因治疗中的应用以及存在的问题进行了阐述,总结了近年来CRISPR系统的设计优化方案,并对该技术的应用前景进行了展望。

Gene editing is a technology of DNA nucleotide sequence modification,which can precisely cut target DNA fragments and insert new gene fragments.It is an important technology in genetic engineering.The emergence of regular clustered short palindrome repetition(CRISPR)technology marks that gene editing technology has entered a new field,which provides great help for the research of gene function and the treatment of diseases.This article states the theory,application and existing problems of CRISPR system,summarizes the design and optimization of CRISPR system in recent years,and prospects the development prospect and trend of gene technology.