Muscle Gene Therapy

  • Dongsheng¬†Duan

Table of contents

  1. Front Matter
    Pages i-xv
  2. Bruce F. Smith, Roberta Wrighten
    Pages 1-21
  3. Bhanu Munil Koppanati, Paula R. Clemens
    Pages 23-40
  4. Gawiyou Danialou, Basil J. Petrof
    Pages 41-53
  5. Chunping Qiao, Xiao Xiao
    Pages 55-68
  6. Annemieke Aartsma-Rus, Gert-Jan B van Ommen
    Pages 69-84
  7. Lindsay M. Wallace, Sara E. Garwick, Scott Q. Harper
    Pages 99-115
  8. Catherine E. Winbanks, Paul Gregorevic
    Pages 117-139
  9. Jin-Hong Shin, Brian Bostick, Deborah M. Fine, Yongping Yue, Dongsheng Duan
    Pages 141-162
  10. Dilip Garikipati, Jeffrey S. Chamberlain
    Pages 163-179
  11. Yi Lai, Yongping Yue, Brian Bostick, Dongsheng Duan
    Pages 205-218
  12. Cathryn S. Mah, Christina A. Pacak, Barry J. Byrne
    Pages 219-230
  13. Yan Zhi, Guangping Gao
    Pages 231-247
  14. Mirella Meregalli, Andrea Farini, Yvan Torrente
    Pages 249-256
  15. Back Matter
    Pages 269-275

About this book


Muscle disease represents an important health threat to the general population. For thousands of years, a cure has been deemed extremely remote, if not impossible, for many relentless muscle diseases such as Duchenne muscular dystrophy. The cloning of muscle disease genes and the prospect of introducing the normal gene to diseased muscle by gene therapy finally bring the hope of a cure. Tremendous progress has been made chasing the muscle gene therapy dream over the past two decades. Valuable information is scattered in the literature. This book represents the first compilation specifically dedicated to issues related to muscle gene therapy. It offers a much needed, up-to-date overview and perspective on the current strategies and status of muscle gene therapy. It covers the cutting edge development of a variety of fascinating strategies such as exon-skipping, AAV gene therapy, RNAi and stem cell approaches. With contributions from prominent investigators in the field, it provides a framework to translate bench science to clinical practice in the upcoming years. This book is a must-have for anyone who is interested in muscle gene therapy including established investigators, clinicians, post-doctoral fellows, graduate students, funding agencies, patients and their families and friends.


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

Editors and affiliations

  • Dongsheng¬†Duan
    • 1
  1. 1.Molecular Microbiology & ImmunologyUniversity of MissouriColumbiaU.S.A.

Bibliographic information