Gene Editing of Hematopoietic Stem and Progenitor Cells
WEN Wei, SHI Jun, ZHANG Jianping, CHENG Tao, ZHANG Xiaobing*
The rapid development of gene-editing technologies has opened new opportunities for HSPC (hematopoietic stem and progenitor cell) gene therapies. Gene editing techniques such as CRISPR-Cas9 enable precise modifications at desired gene sites. Currently, it can achieve high on-target editing efficiencies, whereas there are also safety concerns, such as off-target effects and large-deletion mutations. Clinical trials have demonstrated the potential of HSPC gene editing therapies. However, the impairment of HSPC function and genomic integrity after editing deserves attention. This review summarizes the principle of gene editing, the basic and clinical advances in gene editing of HSPC. Furthermore, it also discusses putative strategies to improve the safety profiles of gene editing products and establish feasible quality control processes.
CN
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