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使用人类诱导多能干细胞进行疾病建模的技术挑战。

Technical challenges in using human induced pluripotent stem cells to model disease.

机构信息

The Whitehead Institute for Biomedical Research, Cambridge, MA 02142, USA.

出版信息

Cell Stem Cell. 2009 Dec 4;5(6):584-95. doi: 10.1016/j.stem.2009.11.009.

Abstract

Reprogramming of human somatic cells uses readily accessible tissue, such as skin or blood, to generate embryonic-like induced pluripotent stem cells (iPSCs). This procedure has been applied to somatic cells from patients who are classified into a disease group, thus creating "disease-specific" iPSCs. Here, we examine the challenges and assumptions in creating a disease model from a single cell of the patient. Both the kinetics of disease onset and progression as well as the spatial localization of disease in the patient's body are challenges to disease modeling. New tools in genetic modification, reprogramming, biomaterials, and animal models can be used for addressing these challenges.

摘要

人类体细胞的重编程利用易于获得的组织,如皮肤或血液,来产生类似胚胎的诱导多能干细胞(iPSCs)。该方法已应用于来自患者的体细胞,从而产生“疾病特异性”iPSCs。在这里,我们研究了从患者的单个细胞创建疾病模型所面临的挑战和假设。疾病的发病和进展的动力学以及患者体内疾病的空间定位都是疾病建模的挑战。遗传修饰、重编程、生物材料和动物模型方面的新工具可用于应对这些挑战。

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