清脆的
基因组编辑
转录激活物样效应核酸酶
Cas9
锌指核酸酶
生物医学
遗传增强
计算生物学
效应器
疾病
生物
计算机科学
生物信息学
基因
医学
遗传学
免疫学
病理
作者
Mengying Dong,Jian‐Gen Liu,Caixia Liu,He Wang,Wei Sun,Bin Liu
标识
DOI:10.1016/j.phrs.2022.106480
摘要
The development of gene-editing technology has been one of the biggest advances in biomedicine over the past two decades. Not only can it be used as a research tool to build a variety of disease models for the exploration of disease pathogenesis at the genetic level, it can also be used for prevention and treatment. This is done by intervening with the expression of target genes and carrying out precise molecular targeted therapy for diseases. The simple and flexible clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 gene-editing technology overcomes the limitations of zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs). For this reason, it has rapidly become a preferred method for gene editing. As a new gene intervention method, CRISPR/Cas9 has been widely used in the clinical treatment of tumours and rare diseases; however, its application in the field of cardiovascular diseases is currently limited. This article reviews the application of the CRISPR/Cas9 editing technology in cardiovascular disease research and treatment, and discusses the limitations and prospects of this technology.
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