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Using human pluripotent stem cells to untangle neurodegenerative disease mechanisms

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Abstract

Human pluripotent stem cells, including embryonic (hES) and induced pluripotent stem cells (hiPS), retain the ability to self-renew indefinitely, while maintaining the capacity to differentiate into all cell types of the nervous system. While human pluripotent cell-based therapies are unlikely to arise soon, these cells can currently be used as an inexhaustible source of committed neurons to perform high-throughput screening and safety testing of new candidate drugs. Here, we describe critically the available methods and molecular factors that are used to direct the differentiation of hES or hiPS into specific neurons. In addition, we discuss how the availability of patient-specific hiPS offers a unique opportunity to model inheritable neurodegenerative diseases and untangle their pathological mechanisms, or to validate drugs that would prevent the onset or the progression of these neurological disorders.

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Authors and Affiliations

  1. GIGA-Neurosciences, University of Liege, Liege, Belgium

    Brigitte Malgrange, Laurence Borgs, Benjamin Grobarczyk, Audrey Purnelle, Patricia Ernst, Gustave Moonen & Laurent Nguyen

  2. Department of Neurology, C.H.U. Sart Tilman, Liege, Belgium

    Gustave Moonen

  3. 1 Avenue de l’Hôpital, B36, Tour de Pharmacie +1, 4000, Liege, Belgium

    Brigitte Malgrange

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  1. Brigitte Malgrange
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Malgrange, B., Borgs, L., Grobarczyk, B. et al. Using human pluripotent stem cells to untangle neurodegenerative disease mechanisms. Cell. Mol. Life Sci. 68, 635–649 (2011). https://doi.org/10.1007/s00018-010-0557-6

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