清脆的
基因组编辑
诱导多能干细胞
生物
Cas9
体细胞
锌指核酸酶
基因组
遗传增强
基因
计算生物学
遗传学
胚胎干细胞
作者
Yan Chen,Ruiting Wen,Zhigang Yang,Zhanghui Chen
出处
期刊:Gene Therapy
[Springer Nature]
日期:2021-03-09
卷期号:29 (5): 207-216
被引量:10
标识
DOI:10.1038/s41434-021-00247-9
摘要
The clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9) system is a versatile and convenient genome-editing tool with prospects in gene therapy. This technique is based on customized site-specific nucleases with programmable guiding RNAs that cleave and introduce double-strand breaks (DSBs) at the target locus and achieve precise genome modification by triggering DNA repair mechanisms. Human hematopoietic stem/progenitor cells (HSPCs) are conventional cell targets for gene therapy in hematological diseases and have been widely used in most studies. Induced pluripotent stem cells (iPSCs) can be generated from a variety of somatic cells and hold great promise for personalized cell-based therapies. CRISPR/Cas9-mediated genome editing in autologous HSPCs and iPSCs is an ideal therapeutic solution for treating hereditary hematological disorders. Here, we review and summarize the latest studies about CRISPR/Cas9-mediated genome editing in patient-derived HSPCs and iPSCs to treat hereditary hematological disorders. Current challenges and prospects are also discussed.
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