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Mesenchymal Stem Cells for Trinucleotide Repeat Disorders

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Trinucleotide Repeat Protocols

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1010))

Abstract

Mesenchymal stem cells/marrow stromal cells (MSCs) are ideally suited for cellular therapy due to their ease of isolation, manipulation, and strong safety profile in the clinic. They can be expanded from normal qualified human donors in large quantities and can be infused without tissue matching, since they shield themselves from the immune system. The ability to be transplanted without tissue matching has allowed large multicenter trials to be conducted with direct comparison of the same batches of MSCs, without adverse events or rejection reactions. MSCs are now approved as drugs in several countries outside of the USA. MSCs can be genetically modified to provide sustained and long-term delivery of growth factors at supraphysiological levels. Gene-modified MSCs are in clinical trials for the treatment of stroke and are under consideration for the treatment of neurodegenerative disorders such as Huntington’s disease.

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Notes

  1. 1.

    The lots of sera must be screened for optimal MSC growth.

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Author information

Authors and Affiliations

  1. Stem Cell Program, Department of Internal Medicine, University of California, Davis, Sacramento, CA, USA

    Geralyn Annett, Gerhard Bauer & Jan A. Nolta

Authors
  1. Geralyn Annett
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  2. Gerhard Bauer
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  3. Jan A. Nolta
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Editor information

Editors and Affiliations

  1. Life Sciences Division, Lawrence Berkeley National Laboratory, Cyclotron Road, MS-977 1, Berkeley, 94720, California, USA

    Yoshinori Kohwi

  2. Life Sciences Division, Lawrence Berkeley National Laboratory, Cyclotron Road, MS-83 1, Berkeley, 94720, California, USA

    Cynthia T. McMurray

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© 2013 Springer New York

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Annett, G., Bauer, G., Nolta, J.A. (2013). Mesenchymal Stem Cells for Trinucleotide Repeat Disorders. In: Kohwi, Y., McMurray, C. (eds) Trinucleotide Repeat Protocols. Methods in Molecular Biology, vol 1010. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-62703-411-1_6

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  • DOI: https://doi.org/10.1007/978-1-62703-411-1_6

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  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-62703-410-4

  • Online ISBN: 978-1-62703-411-1

  • eBook Packages: Springer Protocols

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