Disease Models from Pluripotent Stem Cells Turning Back Time in Disease Pathogenesis?
Disease Models from Pluripotent Stem Cells Turning Back Time in Disease Pathogenesis?
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DOI：
10.1111/j.1749-6632.2009.04962.x
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发表时间：
2009-01-01
期刊：
影响因子：
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通讯作者：
Daley, George Q.
中科院分区：
文献类型：
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作者：
Lengerke, Claudia;Daley, George Q.
先天性和获得性疾病的小鼠模型是无价的，但往往不能忠实地反映人类病理生理。体外分化的胚胎干（ES）细胞概括了早期胚胎发生的各个方面，并分化成多个体细胞组织，从而为人类发育研究提供了一个强大的平台。对基因修饰的胚胎干细胞的分析（例如，通过慢病毒基因转导或从携带遗传疾病的胚胎中提取）提供了前所未有的机会，可以详细研究胚胎发育过程中的疾病发生和进展。通过体细胞重编程从各种疾病患者身上获得的胚胎干细胞和诱导多能干细胞（iPS）有望对疾病的渐进发病机制提供独特的见解，并且能够通过在自体细胞中进行体外基因校正来开发定制的细胞疗法。
Murine models of congenital and acquired diseases are invaluable yet often do not faithfully mirror human pathophysiology. Embryonic stem (ES) cells differentiated in vitro recapitulate aspects of early embryogenesis and differentiate into multiple somatic tissues, thereby serving as a powerful platform for developmental studies in the human. Analysis of genetically modified ES cells (by lentiviral gene transduction or derivation from embryos carrying genetic diseases, for example) offers the unprecedented opportunity to study in detail disease initiation and progression during embryonic development. ES cells and induced pluripotent stem (iPS) cells obtained by somatic cell reprogramming from patients affected by various disorders promise unique insights into the gradual pathogenesis of disease, moreover enabling development of customized cellular therapies by in vitro gene correction in autologous cells.