Article
Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.
Scientific reports - 11 Mar 2015
Betts Corinne A, Saleh Amer F, Carr Carolyn A, Hammond Suzan M, Coenen-Stass Anna M L, Godfrey Caroline, McClorey Graham, Varela Miguel A, Roberts Thomas C, Clarke Kieran, Gait Michael J, Wood Matthew J A
Abstract excerpt
Duchenne muscular dystrophy (DMD) is a fatal neuromuscular disorder caused by mutations in the Dmd gene. In addition to skeletal muscle wasting, DMD patients develop cardiomyopathy, which significantly contributes to mortality. Antisense oligonucleotides (AOs) are a promising DMD therapy, restoring functional dystrophin protein by exon skipping. However, a major limitation with current AOs is the absence of...
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