Laboratory of Molecular Cell Biology, Institute of Biochemistry and Cell Biology, Shanghai Institutes for Biological Sciences, Chinese Academy for Sciences, Yueyang Road 320, 200031 Shanghai, China.
Nature. 2011 May 11;475(7356):386-9. doi: 10.1038/nature10116.
The generation of functional hepatocytes independent of donor liver organs is of great therapeutic interest with regard to regenerative medicine and possible cures for liver disease. Induced hepatic differentiation has been achieved previously using embryonic stem cells or induced pluripotent stem cells. Particularly, hepatocytes generated from a patient's own induced pluripotent stem cells could theoretically avoid immunological rejection. However, the induction of hepatocytes from induced pluripotent stem cells is a complicated process that would probably be replaced with the arrival of improved technology. Overexpression of lineage-specific transcription factors directly converts terminally differentiated cells into some other lineages, including neurons, cardiomyocytes and blood progenitors; however, it remains unclear whether these lineage-converted cells could repair damaged tissues in vivo. Here we demonstrate the direct induction of functional hepatocyte-like (iHep) cells from mouse tail-tip fibroblasts by transduction of Gata4, Hnf1α and Foxa3, and inactivation of p19(Arf). iHep cells show typical epithelial morphology, express hepatic genes and acquire hepatocyte functions. Notably, transplanted iHep cells repopulate the livers of fumarylacetoacetate-hydrolase-deficient (Fah(-/-)) mice and rescue almost half of recipients from death by restoring liver functions. Our study provides a novel strategy to generate functional hepatocyte-like cells for the purpose of liver engineering and regenerative medicine.
不依赖供体肝脏器官生成功能性肝细胞对于再生医学和肝脏疾病的可能治疗具有重要的治疗意义。先前已经使用胚胎干细胞或诱导多能干细胞实现了诱导性肝分化。特别是,来自患者自身诱导多能干细胞的肝细胞在理论上可以避免免疫排斥。然而,从诱导多能干细胞诱导产生肝细胞是一个复杂的过程,随着技术的改进,该过程可能会被取代。谱系特异性转录因子的过表达可直接将终末分化细胞转化为其他一些谱系,包括神经元、心肌细胞和血液祖细胞;然而,这些谱系转换细胞是否能在体内修复受损组织尚不清楚。在这里,我们通过转导 Gata4、Hnf1α 和 Foxa3 以及失活 p19(Arf),从小鼠尾尖成纤维细胞中直接诱导产生功能性肝细胞样(iHep)细胞。iHep 细胞表现出典型的上皮形态,表达肝基因并获得肝细胞功能。值得注意的是,移植的 iHep 细胞可以在 fumarylacetoacetate-hydrolase-deficient(Fah(-/-))小鼠中重新填充肝脏,并通过恢复肝脏功能挽救近一半的受者免于死亡。我们的研究为生成功能性肝细胞样细胞提供了一种新策略,用于肝脏工程和再生医学。