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Genes 2017, 8(4), 108; doi:10.3390/genes8040108

Dystrophic Cardiomyopathy—Potential Role of Calcium in Pathogenesis, Treatment and Novel Therapies

1
School of Human Sciences, The University of Western Australia, Crawley, WA 6009, Australia
2
Victor Chang Cardiac Research Institute, Sydney, NSW 2010, Australia
*
Author to whom correspondence should be addressed.
Academic Editor: Selvarangan Ponnazhagan
Received: 28 November 2016 / Revised: 16 March 2017 / Accepted: 21 March 2017 / Published: 24 March 2017
(This article belongs to the Special Issue Therapeutic Alternative Splicing: Mechanisms and Applications)
View Full-Text   |   Download PDF [6851 KB, uploaded 24 March 2017]   |  

Abstract

Duchenne muscular dystrophy (DMD) is caused by defects in the DMD gene and results in progressive wasting of skeletal and cardiac muscle due to an absence of functional dystrophin. Cardiomyopathy is prominent in DMD patients, and contributes significantly to mortality. This is particularly true following respiratory interventions that reduce death rate and increase ambulation and consequently cardiac load. Cardiomyopathy shows an increasing prevalence with age and disease progression, and over 95% of patients exhibit dilated cardiomyopathy by the time they reach adulthood. Development of the myopathy is complex, and elevations in intracellular calcium, functional muscle ischemia, and mitochondrial dysfunction characterise the pathophysiology. Current therapies are limited to treating symptoms of the disease and there is therefore an urgent need to treat the underlying genetic defect. Several novel therapies are outlined here, and the unprecedented success of phosphorodiamidate morpholino oligomers (PMOs) in preclinical and clinical studies is overviewed. View Full-Text
Keywords: duchenne muscular dystrophy; L-type calcium channel; mitochondria; calcium; cytoskeleton; molecular therapeutics; cardiomyopathy duchenne muscular dystrophy; L-type calcium channel; mitochondria; calcium; cytoskeleton; molecular therapeutics; cardiomyopathy
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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Johnstone, V.P.A.; Viola, H.M.; Hool, L.C. Dystrophic Cardiomyopathy—Potential Role of Calcium in Pathogenesis, Treatment and Novel Therapies. Genes 2017, 8, 108.

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