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诱导多能干细胞研究:应对药物筛选挑战的革命性方法。

Induced pluripotent stem cell research: a revolutionary approach to face the challenges in drug screening.

机构信息

Department of Animal Biotechnology, Animal Resources Research Center, and SMART-IABS, Konkuk University, Seoul 143-701, Korea.

出版信息

Arch Pharm Res. 2012 Feb;35(2):245-60. doi: 10.1007/s12272-012-0205-9. Epub 2012 Feb 28.

DOI:10.1007/s12272-012-0205-9
PMID:22370779
Abstract

Discovery of induced pluripotent stem (iPS) cells in 2006 provided a new path for cell transplantation and drug screening. The iPS cells are stem cells derived from somatic cells that have been genetically reprogrammed into a pluripotent state. Similar to embryonic stem (ES) cells, iPS cells are capable of differentiating into three germ layers, eliminating some of the hurdles in ES cell technology. Further progress and advances in iPS cell technology, from viral to non-viral systems and from integrating to non-integrating approaches of foreign genes into the host genome, have enhanced the existing technology, making it more feasible for clinical applications. In particular, advances in iPS cell technology should enable autologous transplantation and more efficient drug discovery. Cell transplantation may lead to improved treatments for various diseases, including neurological, endocrine, and hepatic diseases. In studies on drug discovery, iPS cells generated from patient-derived somatic cells could be differentiated into specific cells expressing specific phenotypes, which could then be used as disease models. Thus, iPS cells can be helpful in understanding the mechanisms of disease progression and in cell-based efficient drug screening. Here, we summarize the history and progress of iPS cell technology, provide support for the growing interest in iPS cell applications with emphasis on practical uses in cell-based drug screening, and discuss some challenges faced in the use of this technology.

摘要

2006 年诱导多能干细胞(iPS)的发现为细胞移植和药物筛选提供了新途径。iPS 细胞是从体细胞中提取的干细胞,这些体细胞经过基因重编程而具有多能性。与胚胎干细胞(ES)类似,iPS 细胞能够分化为三个胚层,消除了 ES 细胞技术中的一些障碍。iPS 细胞技术的进一步发展和进步,从病毒到非病毒系统,从整合到非整合外源基因到宿主基因组的方法,都增强了现有技术,使其更适合临床应用。特别是,iPS 细胞技术的进步应该能够实现自体移植和更有效的药物发现。细胞移植可能会为各种疾病的治疗带来改善,包括神经、内分泌和肝脏疾病。在药物发现的研究中,从患者来源的体细胞中产生的 iPS 细胞可以分化为表达特定表型的特定细胞,然后可以将这些细胞用作疾病模型。因此,iPS 细胞有助于了解疾病进展的机制,并进行基于细胞的高效药物筛选。在这里,我们总结了 iPS 细胞技术的历史和进展,为 iPS 细胞应用的日益增长的兴趣提供了支持,重点介绍了在基于细胞的药物筛选中的实际应用,并讨论了使用这项技术所面临的一些挑战。

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Induced pluripotent stem cell research: a revolutionary approach to face the challenges in drug screening.诱导多能干细胞研究:应对药物筛选挑战的革命性方法。
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