Summary and Future Directions

Despite major technical and biological obstacles, several promising methods for achieving effective respiratory muscle gene transfer have emerged over the past few years. These developments are exciting and raise a real prospect for gene therapy of the respiratory muscles in the not too distant future. A major challenge is to now refine and adapt these methods to large animal models which are more relevant and analogous to the physiological scale encountered in human patients. Further advances in minimally invasive surgical techniques for targeting the diaphragm and its circulatory system, as well as ongoing developments in vector biology and improvements in our understanding of the human immune response to gene therapy interventions, will also be essential in this effort.

Acknowledgment 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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