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Induced Pluripotent Stem Cells for Disease Modeling and Drug Discovery in Neurodegenerative Diseases

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Abstract

Although most neurodegenerative diseases have been closely related to aberrant accumulation of aggregation-prone proteins in neurons, understanding their pathogenesis remains incomplete, and there is no treatment to delay the onset or slow the progression of many neurodegenerative diseases. The availability of induced pluripotent stem cells (iPSCs) in recapitulating the phenotypes of several late-onset neurodegenerative diseases marks the new era in in vitro modeling. The iPSC collection represents a unique and well-characterized resource to elucidate disease mechanisms in these diseases and provides a novel human stem cell platform for screening new candidate therapeutics. Modeling human diseases using iPSCs has created novel opportunities for both mechanistic studies as well as for the discovery of new disease therapies. In this review, we introduce iPSC-based disease modeling in neurodegenerative diseases, such as Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and amyotrophic lateral sclerosis. In addition, we discuss the implementation of iPSCs in drug discovery associated with some new techniques.

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Acknowledgments

This work was supported by grants from the National Natural Science Foundation of China to L.T. (81171209 and 81371406) and J.T.Y. (81000544), grants from the Shandong Provincial Natural Science Foundation to L.T. (ZR2011HZ001) and J.T.Y. (ZR2010HQ004), the Medicine and Health Science Technology Development Project of Shandong Province to L.T. (2011WSA02018) and J.T.Y. (2011WSA02020), and the Innovation Project for Postgraduates of Jiangsu Province to T.J. (CXLX13_561).

Conflict of Interest

The authors declare no conflict of interest.

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Authors and Affiliations

  1. Department of Neurology, Qingdao Municipal Hospital, Nanjing Medical University, Nanjing, China

    Lei Cao, Lan Tan, Teng Jiang, Xi-Chen Zhu & Jin-Tai Yu

  2. Department of Neurology, Qingdao Municipal Hospital, School of Medicine, Qingdao University, Qingdao, People’s Republic of China

    Lan Tan & Jin-Tai Yu

  3. Department of Neurology, Qingdao Municipal Hospital, College of Medicine and Pharmaceutics, Ocean University of China, Qingdao, China

    Lan Tan & Jin-Tai Yu

  4. Memory and Aging Center, Department of Neurology, University of California, San Francisco, CA, USA

    Jin-Tai Yu

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  1. Lei Cao
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  2. Lan Tan
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  4. Xi-Chen Zhu
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Correspondence to Lan Tan or Jin-Tai Yu.

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Cao, L., Tan, L., Jiang, T. et al. Induced Pluripotent Stem Cells for Disease Modeling and Drug Discovery in Neurodegenerative Diseases. Mol Neurobiol 52, 244–255 (2015). https://doi.org/10.1007/s12035-014-8867-6

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