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Use of Glycine to Augment Exon Skipping and Cell Therapies for Duchenne Muscular Dystrophy

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Muscular Dystrophy Therapeutics

Part of the book series: Methods in Molecular Biology ((MIMB,volume 2587))

Abstract

Antisense oligonucleotide (AO)-based exon-skipping and cell therapies are the main therapeutic approaches for Duchenne muscular dystrophy (DMD). Insufficient systemic delivery leading to low therapeutic efficacy limits the former; low transplantation efficiency hampers the latter. Here we describe how glycine can address these issues by augmenting satellite proliferation and muscle regeneration, resulting in enhanced AO uptake in regenerating myofibers and cell transplantation efficiency in dystrophic mice. The dual functionality of glycine demonstrated in AO-based exon-skipping and cell therapies presents a simple and efficient method to augment AO potency and cell transplantation efficacy in DMD and other muscle diseases.

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Author information

Authors and Affiliations

  1. School of Medical Laboratory & Department of Cell Biology, Tianjin Medical University, Tianjin, China

    Gang Han, Caorui Lin & HaiFang Yin

Authors
  1. Gang Han
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  2. Caorui Lin
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  3. HaiFang Yin
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Corresponding authors

Correspondence to Gang Han or HaiFang Yin .

Editor information

Editors and Affiliations

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

    Rika Maruyama

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

    Toshifumi Yokota

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© 2023 The Author(s), under exclusive license to Springer Science+Business Media, LLC, part of Springer Nature

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Han, G., Lin, C., Yin, H. (2023). Use of Glycine to Augment Exon Skipping and Cell Therapies for Duchenne Muscular Dystrophy. In: Maruyama, R., Yokota, T. (eds) Muscular Dystrophy Therapeutics. Methods in Molecular Biology, vol 2587. Humana, New York, NY. https://doi.org/10.1007/978-1-0716-2772-3_10

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  • DOI: https://doi.org/10.1007/978-1-0716-2772-3_10

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  • Publisher Name: Humana, New York, NY

  • Print ISBN: 978-1-0716-2771-6

  • Online ISBN: 978-1-0716-2772-3

  • eBook Packages: Springer Protocols

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