Muscle Gene Therapy

Methods and Protocols

  • Dongsheng Duan

Part of the Methods in Molecular Biology book series (MIMB, volume 709)

Table of contents

  1. Front Matter
    Pages i-xiii
  2. Basic Methodology Related to Muscle Gene Therapy

    1. Front Matter
      Pages 1-1
    2. Charis L. Himeda, Xiaolan Chen, Stephen D. Hauschka
      Pages 3-19
    3. Takis Athanasopoulos, Helen Foster, Keith Foster, George Dickson
      Pages 21-37
    4. Chady H. Hakim, Dejia Li, Dongsheng Duan
      Pages 75-89
    5. Brian Bostick, Yongping Yue, Dongsheng Duan
      Pages 91-104
    6. Joe N. Kornegay, Janet R. Bogan, Daniel J. Bogan, Martin K. Childers, Robert W. Grange
      Pages 105-123
  3. New Technology in Muscle Gene Therapy

  4. Methods for Muscle Gene Transfer in Large Animal Models

    1. Front Matter
      Pages 237-237
    2. Khalil N. Abi-Nader, Anna L. David
      Pages 239-256
    3. Angela M. Bodles-Brakhop, Ruxandra Draghia-Akli, Kate Broderick, Amir S. Khan
      Pages 257-264
    4. Mihail Petrov, Alock Malik, Andrew Mead, Charles R. Bridges, Hansell H. Stedman
      Pages 277-286
    5. Louise R. Rodino-Klapac, Chrystal L. Montgomery, Jerry R. Mendell, Louis G. Chicoine
      Pages 287-298
    6. Toshifumi Yokota, Eric Hoffman, Shin’ichi Takeda
      Pages 299-312
    7. Yongping Yue, Jin-Hong Shin, Dongsheng Duan
      Pages 313-329
    8. JaBaris D. Swain, Michael G. Katz, Jennifer D. White, Danielle M. Thesier, Armen Henderson, Hansell H. Stedman et al.
      Pages 331-354
    9. Yoshiaki Kawase, Dennis Ladage, Roger J. Hajjar
      Pages 355-367
  5. Back Matter
    Pages 379-382

About this book


Gene therapy offers many conceptual advantages to treat muscle diseases, especially various forms of muscular dystrophies; however, it faces a number of unique challenges, including the need to deliver a therapeutic vector to all muscles throughout the body. In Muscle Gene Therapy: Methods and Protocols, expert researchers in the field present a collection of techniques aimed at bridging the translational gap in muscle gene therapy between the prevalent rodent models and vitally important larger animal models. Divided into three sections, this volume examines basic protocols for optimizing the muscle gene expression cassette and for evaluating the therapeutic outcomes, new developments in muscle gene therapy technology such as adeno-associated viral vector (AAV), oligonucleotide-mediated exon-skipping, and novel RNA-based strategies, and step-by-step guidance on muscle gene delivery in swine, ovine, canine, and non-human primates. 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, detailed, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Muscle Gene Therapy: Methods and Protocols serves as an invaluable resource for graduate students, post-doctoral fellows, and principle investigators pursuing the crucial advancement of muscle disease gene therapy in the hope of someday curing these debilitating disorders.


AAV Adeno-associated viral vectors Delivery systems Exon-skipping Gene expression cassette Gene mutations Large animal models Muscular degeneration

Editors and affiliations

  • Dongsheng Duan
    • 1
  1. 1., Dept. of Molecular MicrobiologyUniversity of MissouriColumbiaUSA

Bibliographic information

  • DOI
  • Copyright Information Springer Science+Business Media, LLC 2011
  • Publisher Name Humana Press
  • eBook Packages Springer Protocols
  • Print ISBN 978-1-61737-981-9
  • Online ISBN 978-1-61737-982-6
  • Series Print ISSN 1064-3745
  • Series Online ISSN 1940-6029
  • Buy this book on publisher's site