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诱导多能干细胞在肝脏疾病中的应用。

Application of Induced Pluripotent Stem Cells in Liver Diseases.

作者信息

Yu Yue, Wang Xuehao, Nyberg Scott L

机构信息

Key Laboratory of Living Donor Liver Transplantation, Ministry of Public Health, China; †Liver Transplantation Center, The First Affiliated Hospital of Nanjing Medical University, Nanjing, Jiangsu Province, China.

‡ Division of Experimental Surgery, Mayo Clinic College of Medicine , Rochester, MN , USA.

出版信息

Cell Med. 2014 Apr 22;7(1):1-13. doi: 10.3727/215517914X680056. eCollection 2014 Nov 5.

Abstract

Tens of millions of patients are affected by liver disease worldwide. Many of these patients can benefit from therapy involving hepatocyte transplantation. Liver transplantation is presently the only proven treatment for many medically refractory liver diseases including end-stage liver failure and inherited metabolic liver disease. However, the shortage in transplantable livers prevents over 40% of listed patients per year from receiving a liver transplant; many of these patients die before receiving an organ offer or become too sick to transplant. Therefore, new therapies are needed to supplement whole-organ liver transplantation and reduce mortality on waiting lists worldwide. Furthermore, the remarkable regenerative capacity of hepatocytes in vivo is exemplified by the increasing number of innovative cell-based therapies and animal models of human liver disorders. Induced pluripotent stem cells (iPSCs) have similar properties to those of embryonic stem cells (ESCs) but bypass the ethical concerns of embryo destruction. Therefore, generation of hepatocyte-like cells (HLCs) using iPSC technology may be beneficial for the treatment of severe liver diseases, screening of drug toxicities, basic research of several hepatocytic disorders, and liver transplantation. Here we briefly summarize the growing number of potential applications of iPSCs for treatment of liver disease.

摘要

全球数以千万计的患者受到肝脏疾病的影响。这些患者中有许多人可以从涉及肝细胞移植的治疗中获益。肝移植目前是治疗包括终末期肝衰竭和遗传性代谢性肝病在内的许多药物难治性肝病的唯一经证实的疗法。然而,可移植肝脏的短缺每年使超过40%的在册患者无法接受肝移植;这些患者中有许多人在获得器官供体之前死亡,或者病情太重而无法进行移植。因此,需要新的疗法来补充全器官肝移植,并降低全球等待名单上患者的死亡率。此外,体内肝细胞显著的再生能力体现在越来越多基于细胞的创新疗法和人类肝脏疾病动物模型中。诱导多能干细胞(iPSC)具有与胚胎干细胞(ESC)相似的特性,但避免了胚胎破坏所涉及的伦理问题。因此,利用iPSC技术生成肝细胞样细胞(HLC)可能有益于治疗严重肝病、药物毒性筛查、几种肝细胞疾病的基础研究以及肝移植。在此,我们简要总结iPSC在肝病治疗中越来越多的潜在应用。

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