© 2019

Muscle Gene Therapy

  • Dongsheng Duan
  • Jerry R. Mendell

Table of contents

  1. Front Matter
    Pages i-xvi
  2. Foundations for Muscle Gene Therapy

    1. Front Matter
      Pages 1-1
    2. Paul M. L. Janssen, Jonathan P. Davis
      Pages 3-12
    3. Ning Liu, Rhonda Bassel-Duby
      Pages 13-39
    4. Stephanie M. Shrader, Roberta Wrighten, Bruce F. Smith
      Pages 41-63
    5. Thierry VandenDriessche, Yoke Chin Chai, Dimitri Boon, Marinee K. Chuah
      Pages 81-97
    6. Alessio Rotini, Giorgia Giacomazzi, Ester Sara Di Filippo, Maurilio Sampaolesi
      Pages 99-119
    7. Jenna M. Kastenschmidt, Ali H. Mannaa, Karissa J. Muñoz, S. Armando Villalta
      Pages 121-139
    8. Yi Lai, Dongsheng Duan
      Pages 141-156
    9. Dan Wang, Alexander Brown, Guangping Gao
      Pages 179-192
    10. Michael E. Nance, Dongsheng Duan
      Pages 193-206
    11. Michael W. Lawlor, Joel S. Schneider, Martin K. Childers, Kristy J. Brown
      Pages 207-226
    12. Yetrib Hathout, Kristy J. Brown, Kanneboyina Nagaraju, Eric P. Hoffman
      Pages 239-252
    13. Alan O’Brien, Ronald D. Cohn
      Pages 275-287
  3. Preclinical Muscle Gene Therapy

    1. Front Matter
      Pages 289-289

About this book


About 7 million people worldwide are suffering from various inherited neuromuscular diseases.  Gene therapy brings the hope of treating these diseases at their genetic roots.  Muscle Gene Therapy is the only book dedicated to this topic. The first edition was published in 2010 when the field was just about to enter its prime time.  The progress made since then has been unprecedented.  The number of diseases that have been targeted by gene therapy has increased tremendously.  The gene therapy toolbox is expanded greatly with many creative novel strategies (such as genome editing and therapy with disease-modifying genes).  Most importantly, clinical benefits have begun to emerge in human patients.  To reflect rapid advances in the field, we have compiled the second edition of Muscle Gene Therapy with contributions from experts that have conducted gene therapy studies either in animal models and/or in human patients.  The new edition offers a much needed, up-to-date overview and perspective on the foundation and current status of neuromuscular disease gene therapy.  It provides a framework to the development and regulatory approval of muscle gene therapy drugs in the upcoming years.  This book is a must-have for anyone who is interested in neuromuscular disease gene therapy including those in the research arena (established investigators and trainees in the fields of clinical practice, veterinary medicine and basic biomedical sciences), funding and regulatory agencies, and patient community.


clinical trial development diseases gene gene therapy gene transfer genes muscle vaccine

Editors and affiliations

  • Dongsheng Duan
    • 1
  • Jerry R. Mendell
    • 2
  1. 1.Department of Molecular Microbiology and Immunology, School of Medicine, University of Missouri, Columbia, MO, USA; Department of Neurology, School of Medicine, University of Missouri, Columbia, MO, USADepartment of Biomedical Sciences College of Veterinary Medicine, University of Missouri, Columbia, MO, USA; Department of Bioengineering, University of MissouriColumbiaUSA
  2. 2.Department of Pediatrics Nationwide Children’s Hospital and Research Institute, Department of Neurology Nationwide Children’s Hospital and Research InstituteThe Ohio State UniversityColumbusUSA

About the editors

Dongsheng Duan, Ph.D., is the Margaret Proctor Mulligan Professor in Medical Research, at the Department of Molecular Microbiology & Immunology, Neurology, Bioengineering, and Biomedical Sciences at the University of Missouri.  He received his medical degree from the West China University of Medical Science, Chengdu, China in 1987 and his Ph.D. degree from the University of Pennsylvania, Philadelphia, USA in 1997.  His research in last two decades has focused on the development of adeno-associated virus as a gene therapy vector, pathogenic mechanisms of Duchenne muscular dystrophy, dystrophin biology and preclinical gene therapy for Duchenne muscular dystrophy in murine and canine models.



Jerry R Mendell, M.D. is the Curran-Peters Research Chair and Professor of Pediatrics and Neurology. His career has been devoted to translational clinical science, beginning as a post doctoral fellow at the National Institutes of Health.  His life-long focus has been to make a difference in lives burdened with untreatable neuromuscular disease. He has contributed significantly to the muscular dystrophies and spinal muscular atrophy, at first using pharmaceutical tools and since 1999, the time of his first gene therapy trial, he has been dedicated to the successful replacement of mutant genes that undermine the lives of innocent victims.

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