Gene therapy is an attractive approach being intensively studied to
prevent muscle deterioration in patients with Duchenne muscular
dystrophy. While clinical trials are only in early stages, initial
reports are promising for its effects on ambulation. Cardiopulmonary
failure, however, is the most common cause of mortality in DMD patients,
and little is known regarding the prospects for gene therapy on
alleviating DMD-associated cardiomyopathy and respiratory failure. Here
we review current knowledge regarding effects of gene therapy on DMD
cardiomyopathy and discuss respiratory endpoints that should be
considered as outcome measures in future clinical trials.