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Duchenne Muscular Dystrophy

Methods and Protocols

  • Camilla Bernardini

Part of the Methods in Molecular Biology book series (MIMB, volume 1687)

Table of contents

  1. Front Matter
    Pages i-xii
  2. Reviews and Overview Chapters

  3. Skeletal and Cardiac Muscle in DMD

    1. Front Matter
      Pages 29-29
    2. Sophie I. Mavrogeni, George Markousis-Mavrogenis, Antigoni Papavasiliou, George Papadopoulos, Genovefa Kolovou
      Pages 31-42
    3. Jenna M. Kastenschmidt, Ileen Avetyan, S. A. Villalta
      Pages 43-56
    4. Stephen J. P. Pratt, Shama R. Iyer, Sameer B. Shah, Richard M. Lovering
      Pages 57-72
  4. Omics Approaches

    1. Front Matter
      Pages 73-73
    2. Federica Censi, Giovanni Calcagnini, Eugenio Mattei, Alessandro Giuliani
      Pages 75-89
    3. Sandra Murphy, Kay Ohlendieck
      Pages 91-105
    4. Alexander Morrison-Nozik, Saptarsi M. Haldar
      Pages 107-119
  5. Exon Skipping and Antisense Oligonucleotides (ASOs)

    1. Front Matter
      Pages 121-121
    2. Shouta Miyatake, Yoshitaka Mizobe, Hotake Takizawa, Yuko Hara, Toshifumi Yokota, Shin’ichi Takeda et al.
      Pages 123-141
    3. Takenori Shimo, Rika Maruyama, Toshifumi Yokota
      Pages 143-155
    4. Julie Miro, Cyril F. Bourgeois, Mireille Claustres, Michel Koenig, Sylvie Tuffery-Giraud
      Pages 157-169
    5. Yoichi Negishi, Yuko Ishii, Kei Nirasawa, Eri Sasaki, Yoko Endo-Takahashi, Ryo Suzuki et al.
      Pages 185-192
  6. Biomarkers and Drug Discovery

  7. Last Discoveries and Future Prospectives

    1. Front Matter
      Pages 229-229
    2. Luca Tucciarone, Usue Etxaniz, Martina Sandoná, Silvia Consalvi, Pier Lorenzo Puri, Valentina Saccone
      Pages 231-256
    3. Christine L. Halbert, James M. Allen, Jeffrey S. Chamberlain
      Pages 257-266
  8. Jenna M. Kastenschmidt, Ileen Avetyan, S. A. Villalta
    Pages E1-E1
  9. Back Matter
    Pages 285-287

About this book

Introduction

This volume explores experimental approaches used to study Duchenne muscular dystrophy (DMD), an X-linked degenerative skeletal muscle disease caused by mutations in the dystrophin gene. Including the latest progress and scientific achievements, the book covers recent discoveries achieved through in vivo gene editing which have proven to be promising in restoring dystrophin expression, at least in ameliorating skeletal muscle symptoms, and the contents focus on “Omics” techniques in gene expression, protein expression, miRNAs, and long non-coding RNA analysis, as well as experimental studies of the structural/functional changes affecting the skeletal and cardiac muscles and ongoing preclinical studies and clinical trials. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. 

Authoritative and practical, Duchenne Muscular Dystrophy: Methods and Protocols serves as a guide for researchers exploring the complicated nature of dystrophin in the hope of helping the victims of this disorder.

Keywords

DMD Dystrophin expression Skeletal muscle Exon skipping Drug discovery Targeted molecular treatments Cardiac muscles

Editors and affiliations

  • Camilla Bernardini
    • 1
  1. 1.Istituto di Anatomia Umana e Biologia CellulareUniversità Cattolica del Sacro CuoreRomaItaly

Bibliographic information

  • DOI https://doi.org/10.1007/978-1-4939-7374-3
  • Copyright Information Springer Science+Business Media LLC 2018
  • Publisher Name Humana Press, New York, NY
  • eBook Packages Springer Protocols
  • Print ISBN 978-1-4939-7373-6
  • Online ISBN 978-1-4939-7374-3
  • Series Print ISSN 1064-3745
  • Series Online ISSN 1940-6029
  • Buy this book on publisher's site