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Exon Skipping and Inclusion Therapies pp 231–247Cite as

In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice

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Part of the book series: Methods in Molecular Biology ((MIMB,volume 1828))

Abstract

Dystrophin exon skipping in mdx mice has been the key model for the development of antisense therapy in vivo. Evaluation of exon skipping in this model involves the following two aspects: (1) efficiency and accuracy of exon skipping and levels of dystrophin expression determined by RT-PCR, immunochemistry, and western blotting; (2) therapeutic effects on muscle pathology and functions assessed by histology and functional assays including grip strength measurement, treadmill test, echocardiogram, and hemodynamics for cardiac functions. Here we describe some key considerations and the essential methodologies in detail for exon skipping in mdx mice.

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Acknowledgments

This work was supported by the Carolinas Muscular Dystrophy Research Endowment at the Carolinas HealthCare Foundation.

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Authors and Affiliations

  1. McColl-Lockwood Laboratory for Muscular Dystrophy Research, Department of Neurology, Neuromuscular/ALS Center, Carolinas Medical Center, Charlotte, NC, USA

    Bo Wu, Mingxing Wang, Sapana Shah & Qi Long Lu

Authors
  1. Bo Wu
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  2. Mingxing Wang
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  3. Sapana Shah
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  4. Qi Long Lu
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Corresponding authors

Correspondence to Bo Wu or Qi Long Lu .

Editor information

Editors and Affiliations

  1. Medical Genetics, University of Alberta, Edmonton, AB, Canada

    Toshifumi Yokota

  2. Medical Genetics, University of Alberta, Edmonton, AB, Canada

    Rika Maruyama

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Wu, B., Wang, M., Shah, S., Lu, Q.L. (2018). In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice. In: Yokota, T., Maruyama, R. (eds) Exon Skipping and Inclusion Therapies. Methods in Molecular Biology, vol 1828. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-8651-4_14

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  • DOI: https://doi.org/10.1007/978-1-4939-8651-4_14

  • Published:

  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-8650-7

  • Online ISBN: 978-1-4939-8651-4

  • eBook Packages: Springer Protocols

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