Molecular Neurobiology

, Volume 52, Issue 1, pp 244–255 | Cite as

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

  • Lei Cao
  • Lan TanEmail author
  • Teng Jiang
  • Xi-Chen Zhu
  • Jin-Tai YuEmail author


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.


Chemical genomics Drug screening Drug target High-throughput technologies Induced pluripotent stem cells 



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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Copyright information

© Springer Science+Business Media New York 2014

Authors and Affiliations

  1. 1.Department of Neurology, Qingdao Municipal HospitalNanjing Medical UniversityNanjingChina
  2. 2.Department of Neurology, Qingdao Municipal Hospital, School of MedicineQingdao UniversityQingdaoPeople’s Republic of China
  3. 3.Department of Neurology, Qingdao Municipal Hospital, College of Medicine and PharmaceuticsOcean University of ChinaQingdaoChina
  4. 4.Memory and Aging Center, Department of NeurologyUniversity of CaliforniaSan FranciscoUSA

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