Cardiac consequences to skeletal muscle-centric therapeutics for Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a fatal disease of muscle deterioration. Duchenne muscular dystrophy affects all striated muscles in the body, including the heart. Recent advances in palliative care, largely directed at improving respiratory function, have extended life but paradoxically further unmasked emergent heart disease in DMD patients. New experimental strategies have shown promise in restoring dystrophin in the skeletal muscles of dystrophin- deficient animals. These strategies often have little or no capacity for restitution of dystrophin in the hearts of these animals. This article draws on both clinical data and recent experimental data to posit that effective skeletal muscle restricted therapies for DMD will paradoxically heighten cardiomyopathy and heart failure in these patients.
Townsend
DeWayne
D
Department of Integrative Biology and Physiology, University of Minnesota, Minneapolis, MN 55455, USA. town0045@umn.edu
Yasuda
Soichiro
S
Chamberlain
Jeffrey
J
Metzger
Joseph M
JM
D000818:Animals; D009202:Cardiomyopathies / Q000209:etiology* / Q000378:metabolism / Q000473:pathology; D016189:Dystrophin / Q000096:biosynthesis* / Q000235:genetics; D015316:Genetic Therapy / Q000009:adverse effects*; D006333:Heart Failure / Q000209:etiology* / Q000378:metabolism / Q000473:pathology; D006801:Humans; D018482:Muscle, Skeletal / Q000378:metabolism / Q000473:pathology; D020388:Muscular Dystrophy, Duchenne / Q000150:complications / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D009206:Myocardium / Q000378:metabolism / Q000473:pathology; D018570:Risk Assessment; D012307:Risk Factors; D016896:Treatment Outcome
D016189:Dystrophin