Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders
Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders
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DOI：
10.1038/s41434-021-00247-9
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发表时间：
2021
期刊：
影响因子：
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通讯作者：
Zhang-Hui Chen
中科院分区：
文献类型：
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作者：
Yan Chen;Ruiting Wen;Zhigang Yang;Zhang-Hui Chen
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.
