Expansion ex vivo and clinical applications of hematopoietic stem/progenitor cells

doi:10.3877/cma.j.issn.2095-1221.2024.02.008

Abstract

Abstract:

Hematopoietic stem cells (HSC) are at the top of the blood cell lineage and maintain normal hematopoietic homeostasis and immune function. The clinical application of hematopoietic stem cell transplantation (HSCT) provides the possibility of curing malignant hematological diseases, and it is also an ideal target cell for future gene therapy. There are many challenges with the development of HSC therapy, including poor mobilization of HSCT donors and a limited number of cord blood-derived HSCs. At present, there are still technical challenges in the isolation of HSC ex vivo. Only hematopoietic stem/progenitor cells (HSPC) can be efficiently isolated. HSPC can be expanded ex vivo by adding cytokines, small molecular compounds, simulating HSC survival environment (bone marrow niche) and gene editing intervention, so as to increase the number of HSC to meet clinical needs. Efficient and safe expansion of HSPC ex vivo will promote the development of clinical transplantation applications, which is of great significance for curing blood diseases and providing sufficient number of HSC for gene therapy.

Key words: Hematopoietic stem cells, Ex vivo expansion, Gene Therapy, Hematological malignancies

Yijian Zhang , Long Zhao, Yang Yang, Beibei Zhang, Yiyu Zhang, Bin Zhang. Expansion ex vivo and clinical applications of hematopoietic stem/progenitor cells[J]. Chinese Journal of Cell and Stem Cell(Electronic Edition), 2024, 14(02): 122-127.

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