诱导多能干细胞在血液系统疾病中应用的研究进展

Progress and obstacles of induced pluripotent stem cell on hematologic disease

诱导多能干细胞（iPS细胞）技术通过外源性转录因子的导入可使体细胞直接重构成为胚胎干细胞样的多潜能细胞，在再生医学研究、体外疾病模型建立和药物筛选等方面有广阔的应用前景。近年来，iPS细胞在血液系统疾病中的研究已取得很大进展，包括造血干细胞、造血祖细胞和成熟血细胞的体外诱导，慢性粒细胞白血病疾病模型的建立以应用于研究酪氨酸激酶抑制剂（TKI）的耐药机制，利用iPS细胞通过遗传修饰纠正缺陷基因以治疗单基因遗传性疾病等。但是iPS细胞能否完全替代未经任何修饰的胚胎干细胞，还需要继续深入研究。

Induced pluripotent stem （iPS） cell based on recently developed stem cell reprogramming technique holds great hope for regenerative medicine, in vitro disease modeling and drug evaluation. Recent progress on clinical hematology includes in vitro generation of hematopoietic progenitors and mature blood cells from somatic cells, iPS cells derived from chronic myeloid leukemia cells for the better understanding of the resistance mechanisms of bcr-abl inhibitor imatinib, and moreover, correction the monogenic inherited disease using gene-targeted strategies. However, whether the iPS cells can fully replace human embryonic stem cells still needs further investigation.