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Cells 2016, 5(4), 46;

Translational Prospects and Challenges in Human Induced Pluripotent Stem Cell Research in Drug Discovery

Integrated Technology Research Laboratories, Research Division, Takeda Pharmaceutical Company Limited, 26-1, Muraoka-Higashi 2-chome, Fujisawa, Kanagawa 251-8555, Japan
Authors to whom correspondence should be addressed.
Academic Editor: Alexander E. Kalyuzhny
Received: 24 October 2016 / Revised: 27 November 2016 / Accepted: 15 December 2016 / Published: 21 December 2016
(This article belongs to the Special Issue Ten Years of iPSCs: Current Status and Future Perspectives)
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Despite continuous efforts to improve the process of drug discovery and development, achieving success at the clinical stage remains challenging because of a persistent translational gap between the preclinical and clinical settings. Under these circumstances, the discovery of human induced pluripotent stem (iPS) cells has brought new hope to the drug discovery field because they enable scientists to humanize a variety of pharmacological and toxicological models in vitro. The availability of human iPS cell-derived cells, particularly as an alternative for difficult-to-access tissues and organs, is increasing steadily; however, their use in the field of translational medicine remains challenging. Biomarkers are an essential part of the translational effort to shift new discoveries from bench to bedside as they provide a measurable indicator with which to evaluate pharmacological and toxicological effects in both the preclinical and clinical settings. In general, during the preclinical stage of the drug development process, in vitro models that are established to recapitulate human diseases are validated by using a set of biomarkers; however, their translatability to a clinical setting remains problematic. This review provides an overview of current strategies for human iPS cell-based drug discovery from the perspective of translational research, and discusses the importance of early consideration of clinically relevant biomarkers. View Full-Text
Keywords: iPS cells; drug discovery; translational research; biomarker iPS cells; drug discovery; translational research; biomarker

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Hosoya, M.; Czysz, K. Translational Prospects and Challenges in Human Induced Pluripotent Stem Cell Research in Drug Discovery. Cells 2016, 5, 46.

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