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- Title
Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers†.
- Authors
Jearawiriyapaisarn, Natee; Moulton, Hong M.; Sazani, Peter; Kole, Ryszard; Willis, Monte S.
- Abstract
Aims: The cardiomyopathy found in Duchenne muscular dystrophy (DMD) is responsible for death due to heart failure in ∼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.
- Subjects
CARDIOMYOPATHIES; DUCHENNE muscular dystrophy; HEART failure; DISEASES; DYSTROPHIN
- Publication
Cardiovascular Research, 2010, Vol 85, Issue 3, p444
- ISSN
0008-6363
- Publication type
Article
- DOI
10.1093/cvr/cvp335