Duchenne muscular dystrophy (DMD) is a fatal genetic disease caused by the absence of
the sarcolemmal protein dystrophin. Dilated cardiomyopathy leading to heart failure is a …

Dystrophin deficiency leads to lethal dilated Duchenne cardiomyopathy. A promising
therapy is to deliver a highly abbreviated microdystrophin gene to the heart using adeno …

Duchenne muscular dystrophy (DMD) is a fatal disease of striated muscle deterioration
resulting from the loss of the cytoskeletal protein dystrophin. Most patients develop …

Duchenne muscular dystrophy (DMD) is the most common form of the progressive muscular
dystrophies characterized by defects of the dystrophin gene. Although primarily …

Background—More than 90% of Duchenne muscular dystrophy (DMD) patients develop
cardiomyopathy, and many die of cardiac failure. Despite tremendous progress in skeletal …

Micro-dystrophin gene replacement therapies for Duchenne muscular dystrophy (DMD) are
currently in clinical trials, but have not been thoroughly investigated for their efficacy on …

Abstract Adeno-associated virus (AAV)-mediated microdystrophin gene therapy holds great
promise for treating Duchenne muscular dystrophy (DMD). Previous studies have revealed …

Mutations in the gene encoding for dystrophin leads to structural and functional deterioration
of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy …

Dystrophin plays an important role in muscle contraction, linking the intracellular
cytoskeleton to the extracellular matrix. Mutations of the dystrophin gene leading to a …

Heart disease is a major health threat for Duchenne/Becker muscular dystrophy patients and
carriers. Expression of a 6–8 kb mini-dystrophin gene in the heart holds promise to change …