清脆的
基因组编辑
计算生物学
Cas9
点突变
生物
基础(拓扑)
遗传学
计算机科学
引导RNA
突变
基因
数学
数学分析
作者
Lu Huang,Chao Yang,Yan Chen,Han‐Xiang Deng,Zhiwei Liao,Hongtao Xiao
出处
期刊:Human Gene Therapy
[Mary Ann Liebert]
日期:2023-06-05
卷期号:34 (15-16): 669-681
摘要
Base editing technology, developed from the CRISPR/Cas9 system, is able to efficiently implement single-base substitutions at specific DNA or RNA sites without generating double-strand breaks with precision and efficiency. Point mutations account for 58% of disease-causing genetic mutations in humans, and single nucleotide variants are an important cause of tumorigenesis, and the advent of base editors offers new hope for the study or treatment of such diseases. Although it has some limitations, base editors have been continuously improved in terms of editing efficiency, specificity, and product purity since their development. In this review, we examine the main base editing technologies and discuss their applications and prospects in tumor research and therapy, as well as elaborate on their mode of delivery.
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