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Ex Vivo Expansion of Hematopoietic Stem Cells for Therapeutic Purposes: Lessons from Development and the Niche

1
Department of Immunohematology and Blood Transfusion, L3-Q Leiden University Medical Center, 2333 ZA Leiden, The Netherlands
2
Department of Molecular Cell Biology, Leiden University Medical Center, 2333 ZA Leiden, The Netherlands
3
Batavia Biosciences, Zernikedreef 16, 2333 CL Leiden, The Netherlands
*
Author to whom correspondence should be addressed.
Cells 2019, 8(2), 169; https://doi.org/10.3390/cells8020169
Received: 28 December 2018 / Revised: 8 February 2019 / Accepted: 13 February 2019 / Published: 18 February 2019
(This article belongs to the Special Issue Stem Cells in Personalized Medicine)
Expansion of hematopoietic stem cells (HSCs) for therapeutic purposes has been a “holy grail” in the field for many years. Ex vivo expansion of HSCs can help to overcome material shortage for transplantation purposes and genetic modification protocols. In this review, we summarize improved understanding in blood development, the effect of niche and conservative signaling pathways on HSCs in mice and humans, and also advances in ex vivo culturing protocols of human HSCs with cytokines or small molecule compounds. Different expansion protocols have been tested in clinical trials. However, an optimal condition for ex vivo expansion of human HSCs still has not been found yet. Translating and implementing new findings from basic research (for instance by using genetic modification of human HSCs) into clinical protocols is crucial to improve ex vivo expansion and eventually boost stem cell gene therapy. View Full-Text
Keywords: hematopoietic stem cell; ex vivo expansion; gene therapy; clinics; transplantation hematopoietic stem cell; ex vivo expansion; gene therapy; clinics; transplantation
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Tajer, P.; Pike-Overzet, K.; Arias, S.; Havenga, M.; Staal, F.J. Ex Vivo Expansion of Hematopoietic Stem Cells for Therapeutic Purposes: Lessons from Development and the Niche. Cells 2019, 8, 169.

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