Vo Linda T, Daley George Q
Stem Cell Transplantation Program, Division of Hematology and Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA.
Blood. 2015 Apr 23;125(17):2641-8. doi: 10.1182/blood-2014-10-570234. Epub 2015 Mar 11.
Generating human hematopoietic stem cells (HSCs) from autologous tissues, when coupled with genome editing technologies, is a promising approach for cellular transplantation therapy and for in vitro disease modeling, drug discovery, and toxicology studies. Human pluripotent stem cells (hPSCs) represent a potentially inexhaustible supply of autologous tissue; however, to date, directed differentiation from hPSCs has yielded hematopoietic cells that lack robust and sustained multilineage potential. Cellular reprogramming technologies represent an alternative platform for the de novo generation of HSCs via direct conversion from heterologous cell types. In this review, we discuss the latest advancements in HSC generation by directed differentiation from hPSCs or direct conversion from somatic cells, and highlight their applications in research and prospects for therapy.
当与基因组编辑技术相结合时,从自体组织生成人类造血干细胞(HSCs)是细胞移植治疗以及体外疾病建模、药物发现和毒理学研究的一种有前景的方法。人类多能干细胞(hPSCs)代表了自体组织潜在的无穷供应;然而,迄今为止,从hPSCs进行定向分化产生的造血细胞缺乏强大且持续的多谱系分化潜能。细胞重编程技术是通过从异源细胞类型直接转化从头生成HSCs的另一种平台。在这篇综述中,我们讨论了通过从hPSCs定向分化或从体细胞直接转化来生成HSCs的最新进展,并强调了它们在研究中的应用以及治疗前景。