化学调控CRISPR/Cas9基因编辑技术的研究进展

Recent Advances in Chemical Control of CRISPR/Cas9 Genome Editing Technology

规律间隔成簇短回文重复序列及其相关蛋白9(Clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9,CRISPR/Cas9)基因编辑技术作为一项基因工程领域革新式的技术,为癌症、遗传性疾病及感染性疾病等多种重大疾病的治疗提供了极大的帮助.但如何在特定细胞和组织中实现时空调控的精准基因编辑,进而避免脱靶效应,依然是该技术在临床转化领域面临的重要挑战.近年来,通过化学分子和反应实现对CRISPR/Cas9活性的调控已经成为提升这项基因编辑技术效率的重要手段之一.本文综合评述了一些最近报道的化学调控CRISPR/Cas9基因编辑的方法,并对其在临床医学领域的应用前景进行了展望.

As a revolutionary technology in the field of genetic engineering,clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein 9(CRISPR/Cas9)genome editing contribute significantly to various important diseases,such as cancer,genetic diseases and infectious diseases.However,precisely spatiotemporal control of genome editing in specific cells and tissues to avoid off-target effects is still one of the major challenges for clinical translation of this technology.Recently,using chemical molecules and reactions to control the activity of CRISPR/Cas9 is becoming one of the important means to increase the gene editing efficiency.This review summarized the recent advances about chemical control of CRISPR/Cas9 gene editing and the future applications of these technologies in clinical medicine was proposed.