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Stem Cell-Based Therapies for Polyglutamine Diseases

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Polyglutamine Disorders

Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 1049))

Abstract

Polyglutamine (polyQ) diseases are a family of neurodegenerative disorders with very heterogeneous clinical presentations, although with common features such as progressive neuronal death. Thus, at the time of diagnosis patients might present an extensive and irreversible neuronal death demanding cell replacement or support provided by cell-based therapies. For this purpose stem cells, which include diverse populations ranging from embryonic stem cells (ESCs), to fetal stem cells, mesenchymal stromal cells (MSCs) or induced pluripotent stem cells (iPSCs) have remarkable potential to promote extensive brain regeneration and recovery in neurodegenerative disorders. This regenerative potential has been demonstrated in exciting pre and clinical assays. However, despite these promising results, several drawbacks are hampering their successful clinical implementation. Problems related to ethical issues, quality control of the cells used and the lack of reliable models for the efficacy assessment of human stem cells. In this chapter the main advantages and disadvantages of the available sources of stem cells as well as their efficacy and potential to improve disease outcomes are discussed.

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Authors and Affiliations

  1. Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal

    Liliana S. Mendonça, Isabel Onofre, Catarina Oliveira Miranda, Rita Perfeito, Clévio Nóbrega & Luís Pereira de Almeida

  2. Interdisciplinary Research Institute, University of Coimbra, Coimbra, Portugal

    Liliana S. Mendonça, Catarina Oliveira Miranda & Rita Perfeito

  3. Department of Biomedical Sciences and Medicine, University of Algarve, Faro, Portugal

    Clévio Nóbrega

  4. Centre for Biomedical Research, University of Algarve, Faro, Portugal

    Clévio Nóbrega

  5. Algarve Biomedical Center, University of Algarve, Faro, Portugal

    Clévio Nóbrega

  6. Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal

    Luís Pereira de Almeida

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  1. Liliana S. Mendonça
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  1. Department of Biomedical Sciences and Medicine; Centre for Biomedical Research and Algarve Biomedical Center, University of Algarve, Faro, Portugal

    Clévio Nóbrega

  2. Center for Neuroscience and Cell Biology and Faculty of Pharmacy, University of Coimbra, Coimbra, Portugal

    Luís Pereira de Almeida

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Mendonça, L.S., Onofre, I., Miranda, C.O., Perfeito, R., Nóbrega, C., de Almeida, L.P. (2018). Stem Cell-Based Therapies for Polyglutamine Diseases. In: Nóbrega, C., Pereira de Almeida, L. (eds) Polyglutamine Disorders. Advances in Experimental Medicine and Biology, vol 1049. Springer, Cham. https://doi.org/10.1007/978-3-319-71779-1_21

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