Background The BIO14. 6 hamster is an excellent animal model for inherited
cardiomyopathy, because of its lethal and well-documented course, due to a spontaneous …

Aims δ-Sarcoglycan is a member of the dystrophin-associated glycoprotein complex linking
the cytoskeleton to the extracellular matrix. Similar to patients with defects in the gene …

Background—The success of muscular dystrophy gene therapy requires widespread and
stable gene delivery with minimal invasiveness. Here, we investigated the therapeutic effect …

The BIO14. 6 hamster carries a mutation in the delta sarcoglycan gene causing muscular
dystrophy and cardiomyopathy. The disease can be prevented by systemic delivery of delta …

Intracardiac gene transfer and gene therapy have been investigated with different vector
systems. Here we used adeno-associated virus (AAV) vectors to deliver either a reporter …

Trans-splicing adeno-associated viral (tsAAV) vectors hold great promise for delivering large
therapeutic genes. One potential application is in the treatment of Duchenne muscular …

In humans, a subset of cases of Limb–girdle muscular dystrophy (LGMD) arise from
mutations in the genes encoding one of the sarcoglycan (α, β, γ, or δ) subunits of the …

Recombinant adeno-associated virus (rAAV) vectors allow efficient gene transfer and
expression in the muscle; therefore, rAAVs represent a potential gene therapy vector for …

Heart failure (HF) continues to be a progressive disease with ever increasing morbidity and
mortality in the western world. 1 While progress in conventional treatment modalities is …

The hereditary form comprises≈ 1/5 of patients with dilated cardiomyopathy (DCM) and is a
major cause of advanced heart failure. Medical and socioeconomic settings require novel …