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Pluripotent Stem Cells for Modeling Motor Neuron Diseases

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Recent Advances in Stem Cells

Part of the book series: Stem Cell Biology and Regenerative Medicine ((STEMCELL))

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Abstract

Motoneuron disorders (MNDs) are a group of degenerative diseases characterized by muscular weakness and muscle atrophy that result most often in the death of patients. Today, there is no treatment despite decades of clinical trials. One reason of failures of these trials may be the anatomic and physiologic differences that exist between humans and rodent models of MNDs. With the recent discovery of the human induced pluripotent stem cell (iPSC) technology, new hopes arise to generate human cellular models for MNDs. In this chapter, I will focus on the two most common infantile and adult MNDs, respectively: spinal muscular atrophy (SMA) and amyotrophic lateral sclerosis (ALS). I will describe iPSC models generated from patients with various forms of each disorder and the specific defects observed in iPS-derived motoneurons in order to validate models and to identify new phenotypes that could be targets of future therapies for SMA and ALS patients.

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Authors and Affiliations

  1. Inserm U 1127, CNRS UMR 7225, Sorbonne Universités, UPMC Univ Paris 06 UMR S 1127, Institut du Cerveau et de la Moelle épinière, ICM, Paris, F-75013, France

    Delphine Bohl

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Correspondence to Delphine Bohl .

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  1. Qatar Biomedical Research Institute, Hamad Bin Khalifa University, Doha, Qatar

    Essam M. Abdelalim

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Bohl, D. (2016). Pluripotent Stem Cells for Modeling Motor Neuron Diseases. In: Abdelalim, E. (eds) Recent Advances in Stem Cells. Stem Cell Biology and Regenerative Medicine. Humana Press, Cham. https://doi.org/10.1007/978-3-319-33270-3_5

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