Gene Therapy for the Respiratory Muscles

  • Gawiyou Danialou
  • Basil J. Petrof


The diaphragm and other ventilatory muscles constitute a vital pump for respiration. Death is an inevitable consequence when this pump fails, unless artificial ventilatory support is provided. In many neuromuscular disorders for which gene therapy is being considered (e.g., Duchenne muscular dystrophy), muscle weakness involves the diaphragm and other respiratory muscles, thereby leading to the development of ventilatory failure. Therefore, it is critically important that effective methods be developed for targeting the respiratory muscles by gene therapy approaches. In this chapter, we review the normal physiology of the respiratory muscles and the particular challenges associated with evaluating the efficacy of any future applications of gene therapy to the respiratory muscles in humans. We also review the current state of affairs with respect to preclinical animal models of candidate diseases for respiratory muscle gene therapy, which have pointed to several challenges as well as promising areas for future progress in this area.


Respiratory Muscle Duchenne Muscular Dystrophy Pompe Disease Equine Infectious Anemia Virus Golden Retriever Muscular Dystrophy 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.



This work was supported by grants from the Muscular Dystrophy Association, the Canadian Institutes of Health Research and the Fonds de la recherche en sante du Quebec. The authors have no conflicts of interest to declare.


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© Springer Science+Business Media, LLC 2010

Authors and Affiliations

  1. 1.Meakins-Christie LaboratoriesMcGill UniversityMontrealCanada

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