iPSCs and small molecules: a reciprocal effort towards better approaches for drug discovery

Ru Zhang, Li-hong Zhang, Xin Xie
DOI: 10.1038/aps.2013.21


Ru ZHANG1, Li-hong ZHANG2, Xin XIE1, 2, *
1Laboratory of Receptor-based Bio-medicine, Shanghai Key Laboratory of Signaling and Disease Research, School of Life Sciences and Technology, Tongji University, Shanghai 200092, China; 2CAS Key Laboratory of Receptor Research, the National Center for Drug Screening, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai 201203, China

The revolutionary induced pluripotent stem cell (iPSC) technology provides a new path for cell replacement therapies and drug screening. Patient-specific iPSCs and subsequent differentiated cells manifesting disease phenotypes will finally position human disease pathology at the core of drug discovery. Cells used to test the toxic effects of drugs can also be generated from normal iPSCs and provide a much more accurate and cost-effective system than many animal models. Here, we highlight the recent progress in iPSC-based cell therapy, disease modeling and drug evaluations. In addition, we discuss the use of small molecule drugs to improve the generation of iPSCs and understand the reprogramming mechanism. It is foreseeable that the interplay between iPSC technology and small molecule compounds will push forward the applications of iPSC-based therapy and screening systems in the real world and eventually revolutionize the methods used to treat diseases.

Keywords: induced pluripotent stem cells (iPSCs); disease modeling; drug screening; toxicity evaluation; cell replacement therapy; small molecules; drug development

This work was supported by grants from the Chinese Academy of Sciences (XDA01040301) Ministry of Science and Technology of China (2009CB940900, 2010CB944900, 2011CB965104, 2011DFB30010) and the Shanghai Commission of Science and Technology (11DZ2292200).
* To whom correspondence should be addressed.
Received 2012-12-31 Accepted 2013-02-07

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