Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/<i>mdx</i> Mice.

ABSTRACT: Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disease caused by mutations in the dystrophin gene. DMD boys are wheelchair-bound around 12 years and generally survive into their twenties. There is currently no effective treatment except palliative care, although personalized treatments such as exon skipping, stop codon read-through, and viral-based gene therapies are making progress. Patients present with skeletal muscle pathology, but most also show cardiomyopathy by the age of 10. A systemic therapeutic approach is needed that treats the heart and skeletal muscle defects in all patients. The dystrophin-related protein utrophin has been shown to compensate for the lack of dystrophin in the mildly affected BL10/mdx mouse. The purpose of this investigation was to dem

SUBMITTER: Kennedy TL 

PROVIDER: S-EPMC6216100 | biostudies-literature | 2018 Dec

REPOSITORIES: biostudies-literature