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利用诱导多能干细胞进行疾病建模和细胞治疗:快速安全应用的未来治疗选择。

Disease modeling and cell based therapy with iPSC: future therapeutic option with fast and safe application.

作者信息

Kim Changsung

机构信息

Department of Bioscience and Biotechnology, Sejong University, Seoul, Korea.

出版信息

Blood Res. 2014 Mar;49(1):7-14. doi: 10.5045/br.2014.49.1.7. Epub 2014 Mar 24.

Abstract

Induced pluripotent stem cell (iPSC) technology has shown us great hope to treat various human diseases which have been known as untreatable and further endows personalized medicine for future therapy without ethical issues and immunological rejection which embryonic stem cell (hES) treatment has faced. It has been agreed that iPSCs knowledge can be harnessed from disease modeling which mimics human pathological development rather than trials utilizing conventional rodent and cell lines. Now, we can routinely generate iPSC from patient specific cell sources, such as skin fibroblast, hair follicle cells, patient blood samples and even urine containing small amount of epithelial cells. iPSC has both similarity and dissimilarity to hES. iPSC is similar enough to regenerate tissue and even full organism as ES does, however what we want for therapeutic advantage is limited to regenerated tissue and lineage specific differentiation. Depending on the lineage and type of cells, both tissue memory containing (DNA rearrangement/epigenetics) and non-containing iPSC can be generated. This makes iPSC even better choice to perform disease modeling as well as cell based therapy. Tissue memory containing iPSC from mature leukocytes would be beneficial for curing cancer and infectious disease. In this review, the benefit of iPSC for translational approaches will be presented.

摘要

诱导多能干细胞(iPSC)技术为我们治疗各种曾被认为无法治愈的人类疾病带来了巨大希望,并且为未来的治疗提供了个性化医疗,不存在胚胎干细胞(hES)治疗所面临的伦理问题和免疫排斥。人们一致认为,可以从模拟人类病理发展的疾病模型中获取iPSC知识,而不是利用传统啮齿动物和细胞系进行试验。现在,我们可以常规地从患者特定的细胞来源生成iPSC,如皮肤成纤维细胞、毛囊细胞、患者血液样本,甚至含有少量上皮细胞的尿液。iPSC与hES既有相似之处,也有不同之处。iPSC与ES足够相似,能够像ES一样再生组织甚至完整生物体,然而,我们期望的治疗优势仅限于再生组织和谱系特异性分化。根据细胞的谱系和类型,可以生成含有组织记忆(DNA重排/表观遗传学)的iPSC和不含组织记忆的iPSC。这使得iPSC成为进行疾病建模以及基于细胞的治疗的更佳选择。来自成熟白细胞的含有组织记忆的iPSC将有利于治愈癌症和传染病。在这篇综述中,将阐述iPSC在转化医学方法中的益处。

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