Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.

ABSTRACT:

Aims

The cardiomyopathy found in Duchenne muscular dystrophy (DMD) is responsible for death due to heart failure in approximately 30% of patients and additionally contributes to many DMD morbidities. Strategies to bypass DMD-causing mutations to allow an increase in body-wide dystrophin have proved promising, but increasing cardiac dystrophin continues to be challenging. The purpose of this study was to determine if therapeutic restoration of cardiac dystrophin improved the significant cardiac hypertrophy and diastolic dysfunction identified in X-linked muscular dystrophy (mdx) dystrophin-null mouse due to a truncation mutation over time after treatment.

Methods and results

Mice lacking dystrophin due to a truncation mutation (mdx) were given an arginine-rich, cell-penetrati

SUBMITTER: Jearawiriyapaisarn N 

PROVIDER: S-EPMC2802205 | biostudies-literature | 2010 Feb

REPOSITORIES: biostudies-literature