Abstract Currently, hematopoietic stem cell (HSC) transplantation is widely used in the therapy of hematological malignancies, non-malignant refractory anemia, genetic diseases and certain tumors with satisfactory therapeutic efficacy. HSC sources used for transplantation include bone marrow, mobilized peripheral blood and neonate umbilical cord blood. However, for many patients, sufficient number of human leukocyte antigen (HLA) -matched HSC cannot be found for transplantation, because the number of HSC in these tissues is small and HLA-identical donors are rare. Thus, in vitro generation of HSC has recently been focused. At present, the origin of HSC is hPSC, including hESC and hiPSC, which is worth to be the new origin of HSC transplantation. However, to generate functional hematopoietic stem cells which have efficient multi-lineage differentiation and in vivo engraftment potentials still is a big challenge to be confronted. In this review, the recent technical progress in HSC generation is summarizd, and the problems to be solved and new challenges to be confronted were discussed.
题目: 体外产生可移植的人造血干细胞的研究进展.
摘要: 造血干细胞（hematopoietic stem cell，HSC）移植被广泛应用于恶性血液病、非恶性衰竭性血液疾病以及非血液系统疾病（遗传性疾病和某些实体瘤）的治疗，并取得了良好疗效。临床上常用的移植HSC源有骨髓、动员的外周血及脐带血。但是，由于这些组织中HSC含量很少，及组织相容性抗原特异的供者稀缺等问题，导致许多病人无法获得足够量的组织相容性匹配的HSC。因此，如何通过体外培养获得满足临床需要的HSC已成为近年来学者们研究的热点问题。目前体外产生HSC的主要来源为人多能干细胞（human pluripotent stem cell，hPSC），包括人胚胎干细胞（human embryonic stem cell，hESC）和人诱导多潜能干细胞（human induced pluripotent stem cell，hiPSC），并且有望成为HSC移植的新来源。虽然有研究首次证实通过iPSC可以在体外产生HSC，但是体外产生HSC的功能特性以及其产生的分子机制的探究仍是一个巨大挑战。本文旨在对近年来体外产生人HSC策略和方法的最新研究进展作一综述，并就其存在的问题及面临的挑战展开讨论.