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Muscular Dystrophy Therapeutics

Methods and Protocols

  • Book
  • © 2023

Overview

Editors:
  1. Rika Maruyama

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

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    You can also search for this editor in PubMed   Google Scholar

  2. Toshifumi Yokota

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

    View editor publications

    You can also search for this editor in PubMed   Google Scholar

  • Includes cutting-edge techniques
  • Provides step-by-step detail essential for reproducible results
  • Contains key implementation advice from the experts

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

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Table of contents (31 protocols)

  1. Front Matter

    Pages i-xv
    Download chapter PDF

  2. Basics and Introduction

    1. Front Matter

      Pages 1-1
      Download chapter PDF

    2. Current Strategies of Muscular Dystrophy Therapeutics: An Overview

      • Kenji Rowel Q. Lim, Toshifumi Yokota
      Pages 3-30

    3. Viltolarsen: From Preclinical Studies to FDA Approval

      • Rohini Roy Roshmi, Toshifumi Yokota
      Pages 31-41

  4. Antisense Oligonucleotides and Gene Replacement Therapies

    1. Front Matter

      Pages 105-105
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    2. Restoring Dystrophin Expression by Skipping Exons 6 and 8 in Neonatal Dystrophic Dogs

      • Md Nur Ahad Shah, Toshifumi Yokota
      Pages 107-124

    3. Restoring Dystrophin Expression with Exon 44 and 53 Skipping in the DMD Gene in Immortalized Myotubes

      • Yusuke Echigoya, Toshifumi Yokota
      Pages 125-139

    4. Restoring Dystrophin Expression with Duchenne Muscular Dystrophy Exon 45 Skipping in Induced Pluripotent Stem Cell-Derived Cardiomyocytes

      • Mitsuto Sato, Naoko Shiba, Daigo Miyazaki, Yuji Shiba, Akinori Nakamura
      Pages 141-151

    5. Quantitative Evaluation of Exon Skipping in Urine-Derived Cells for Duchenne Muscular Dystrophy

      • Katsuhiko Kunitake, Chaitra Sathyaprakash, Norio Motohashi, Yoshitsugu Aoki
      Pages 153-164

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

      • Gang Han, Caorui Lin, HaiFang Yin
      Pages 165-182

    7. Morpholino-Mediated Exons 28–29 Skipping of Dysferlin and Characterization of Multiexon-skipped Dysferlin using RT-PCR, Immunoblotting, and Membrane Wounding Assay

      • Saeed Anwar, Toshifumi Yokota
      Pages 183-196

    8. Knocking Down DUX4 in Immortalized Facioscapulohumeral Muscular Dystrophy Patient-Derived Muscle Cells

      • Kenji Rowel Q. Lim, Toshifumi Yokota
      Pages 197-208

    9. Peptide-Conjugated PMOs for the Treatment of Myotonic Dystrophy

      • Jessica Stoodley, David Seoane Miraz, Yahya Jad, Mathieu Fischer, Matthew J. A. Wood, Miguel A. Varela
      Pages 209-237

    10. Developing Therapeutic Splice-Correcting Antisense Oligomers for Adult-Onset Pompe Disease with c.-32-13T>G Mutation

      • Kristin A. Ham, Russell D. Johnsen, Michel Tchan, Steve D. Wilton, May T. Aung-Htut
      Pages 239-251

  5. Gene Replacement Therapies

    1. Front Matter

      Pages 253-253
      Download chapter PDF

    2. Molecular and Biochemical Assessment of Gene Therapy in the Canine Model of Duchenne Muscular Dystrophy

      • Chady H. Hakim, Dennis Pérez-López, Matthew J. Burke, James Teixeira, Dongsheng Duan
      Pages 255-301
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Keywords

About this book

This detailed book presents a comprehensive collection of state-of-the-art protocols on muscular dystrophy therapeutics, covering therapeutics using antisense oligonucleotides, gene replacement, genome editing, small molecules, stem cells, and antibodies. Written by leaders in the field, the volume explores techniques that are currently in use and are starting an exciting therapeutic revolution in muscular dystrophy. As a part of the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step and readily reproducible laboratory protocols, as well as tips on troubleshooting and avoiding known pitfalls. 

Authoritative and practical, Muscular Dystrophy Therapeutics: Methods and Protocols serves as an ideal resource to inspire readers and provide tips, strategies, and advice to develop new therapeutic technologies for this group of diseases.

Editors and Affiliations

  • Department of Medical Genetics, University of Alberta, Edmonton, Canada

    Rika Maruyama, Toshifumi Yokota

Bibliographic Information

  • Book Title: Muscular Dystrophy Therapeutics

  • Book Subtitle: Methods and Protocols

  • Editors: Rika Maruyama, Toshifumi Yokota

  • Series Title: Methods in Molecular Biology

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

  • Publisher: Humana New York, NY

  • eBook Packages: Springer Protocols

  • Copyright Information: The Editor(s) (if applicable) and The Author(s), under exclusive license to Springer Science+Business Media, LLC, part of Springer Nature 2023

  • Hardcover ISBN: 978-1-0716-2771-6Published: 19 November 2022

  • Softcover ISBN: 978-1-0716-2774-7Published: 19 November 2023

  • eBook ISBN: 978-1-0716-2772-3Published: 18 November 2022

  • Series ISSN: 1064-3745

  • Series E-ISSN: 1940-6029

  • Edition Number: 1

  • Number of Pages: XV, 575

  • Number of Illustrations: 48 b/w illustrations, 50 illustrations in colour

  • Topics: Human Genetics, Anatomy, Behavioral Therapy

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