Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China.
Adv Exp Med Biol. 2023;1442:177-199. doi: 10.1007/978-981-99-7471-9_11.
Hematopoietic stem cells (HSCs) can be isolated and collected from the body, genetically modified, and expanded ex vivo. The invention of innovative and powerful gene editing tools has provided researchers with great convenience in genetically modifying a wide range of cells, including hematopoietic stem and progenitor cells (HSPCs). In addition to being used to modify genes to study the functional role that specific genes play in the hematopoietic system, the application of gene editing platforms in HSCs is largely focused on the development of cell-based gene editing therapies to treat diseases such as immune deficiency disorders and inherited blood disorders. Here, we review the application of gene editing tools in HSPCs. In particular, we provide a broad overview of the development of gene editing tools, multiple strategies for the application of gene editing tools in HSPCs, and exciting clinical advances in HSPC gene editing therapies. We also outline the various challenges integral to clinical translation of HSPC gene editing and provide the possible corresponding solutions.
造血干细胞（HSCs）可从体内分离和采集，经过基因修饰后在体外扩增。创新和强大的基因编辑工具的发明为研究人员在广泛的细胞（包括造血干细胞和祖细胞（HSPCs））上进行基因修饰提供了极大的便利。除了用于修饰基因以研究特定基因在造血系统中所起的功能作用外，基因编辑平台在 HSCs 中的应用主要集中在开发基于细胞的基因编辑疗法，以治疗免疫缺陷疾病和遗传性血液疾病等疾病。在这里，我们综述了基因编辑工具在 HSPCs 中的应用。特别是，我们提供了基因编辑工具的发展、多种基因编辑工具在 HSPCs 中的应用策略以及 HSPC 基因编辑疗法的令人兴奋的临床进展的广泛概述。我们还概述了 HSPC 基因编辑临床转化所必需的各种挑战，并提供了可能的相应解决方案。
