Gene therapy is defined by the introduction of new genes or the genetic modification of
existing genes and/or their regulatory portions via gene replacement and gene editing …

Duchenne muscular dystrophy (DMD) is a X-linked, progressive childhood myopathy
caused by mutations in the dystrophin gene, one of the largest genes in the genome. It is …

So far effective strategies to treat cardiomyopathy in patients with muscular dystrophies are
still not clearly defined. Previously, treatment with β-blockers showed beneficial effects on …

The advancement of gene therapy–based approaches to treat heart disease represents a
need for clinically relevant animal models with characteristics equivalent to human …

To establish an efficient, safe immunosuppressive regimen of adeno-associated vector
(AAV)-mediated gene therapy for Duchenne muscular dystrophy (DMD), we evaluated the …

Sarcoglycanopathies are rare autosomic limb girdle muscular dystrophies caused by
mutations in one of the genes coding for sarcoglycans. Sarcoglycans form a complex, which …

Recombinant adeno-associated viral vectors (rAAVs) are used for therapeutic gene transfer
in skeletal muscle, but it is unclear if immune reactivity to gene transfer and persistence of …

Abstract Adeno-associated virus (AAV)-based vectors are promising vehicles for therapeutic
gene delivery, including for the treatment for heart failure. It has been demonstrated for each …

Abstract γ-Sarcoglycanopathy or limb girdle muscular dystrophy type 2C is an untreatable
disease caused by autosomal recessively inherited mutations of the γ-sarcoglycan gene …

Skeletal muscle represents an attractive target tissue for adenoviral gene therapy to treat
muscle disorders and as a production platform for systemic expression of therapeutic …