转录激活物样效应核酸酶
锌指核酸酶
清脆的
基因组编辑
基因组工程
生物
计算生物学
Cas9
遗传学
基因靶向
核酸酶
基因
基因传递
遗传增强
基因转移
作者
Qurrat Ul Ain,Jee Young Chung,Yong Hee Kim
标识
DOI:10.1016/j.jconrel.2014.12.036
摘要
Gene therapy by engineered nucleases is a genetic intervention being investigated for curing the hereditary disorders by targeting selected genes with specific nucleotides for establishment, suppression, abolishment of a function or correction of mutation. Here, we review the fast developing technology of targeted genome engineering using site specific programmable nucleases zinc finger nucleases (ZFNs), transcription activator like nucleases (TALENs) and cluster regulatory interspaced short palindromic repeat/CRISPR associated proteins (CRISPR/Cas) based RNA-guided DNA endonucleases (RGENs) and their different characteristics including pros and cons of genome modifications by these nucleases. We have further discussed different types of delivery methods to induce gene editing, novel development in genetic engineering other than nucleases and future prospects.
科研通智能强力驱动
Strongly Powered by AbleSci AI