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诱导多能干细胞:药物发现的新工具和干细胞治疗的新希望。

Induced pluripotent stem cells, new tools for drug discovery and new hope for stem cell therapies.

机构信息

Division of Neurosciences, and the Center for Gene Expression and Drug Discovery, Beckman Research Institute of City of Hope, 1500 E. Duarte Rd., Duarte, CA 91010, USA.

出版信息

Curr Mol Pharmacol. 2009 Jan;2(1):15-8. doi: 10.2174/1874467210902010015.

Abstract

Somatic cell nuclear transfer or therapeutic cloning has provided great hope for stem cell-based therapies. However, therapeutic cloning has been experiencing both ethical and technical difficulties. Recent breakthrough studies using a combination of four factors to reprogram human somatic cells into pluripotent stem cells without using embryos or eggs have led to an important revolution in stem cell research. Comparative analysis of human induced pluripotent stem cells and human embryonic stem cells using assays for morphology, cell surface marker expression, gene expression profiling, epigenetic status, and differentiation potential have revealed a remarkable degree of similarity between these two pluripotent stem cell types. This mini-review summarizes these ground-breaking studies. These advances in reprogramming will enable the creation of patient-specific stem cell lines to study various disease mechanisms. The cellular models created will provide valuable tools for drug discovery. Furthermore, this reprogramming system provides great potential to design customized patient-specific stem cell therapies with economic feasibility.

摘要

体细胞核移植或治疗性克隆为基于干细胞的治疗提供了巨大的希望。然而,治疗性克隆一直面临着伦理和技术上的困难。最近的突破性研究使用四种因子的组合将人类体细胞重编程为多能干细胞,而不使用胚胎或卵子,这在干细胞研究中引发了一场重要的革命。通过形态学、细胞表面标志物表达、基因表达谱分析、表观遗传状态和分化潜能等检测方法对人诱导多能干细胞和人胚胎干细胞进行比较分析,发现这两种多能干细胞类型之间具有惊人的相似性。这篇综述总结了这些开创性的研究。这些重编程方面的进展将使创建患者特异性干细胞系以研究各种疾病机制成为可能。所创建的细胞模型将为药物发现提供有价值的工具。此外,这种重编程系统为设计具有经济可行性的定制化患者特异性干细胞治疗提供了巨大的潜力。

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