Our Translational Gene Therapy Center has used small molecules for exon skipping and
mutation suppression and gene transfer to replace or provide surrogate genes as tools for …

The muscular dystrophies are disorders of progressive muscular degeneration and
weakness. As a group they display clinical heterogeneity that reflects the heterogeneity of …

Efficient and widespread gene transfer is required for successful treatment of Duchenne
muscular dystrophy (DMD). Here, we performed the first clinical trial using a chimeric adeno …

Mesoangioblasts are stem/progenitor cells derived from a subset of pericytes found in
muscle that express alkaline phosphatase. They have been shown to ameliorate the …

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder resulting from
lesions of the gene encoding dystrophin. These usually consist of large genomic deletions …

No treatment exists for facioscapulohumeral muscular dystrophy (FSHD), one of the most
common inherited muscle diseases. Although FSHD can be debilitating, little effort has been …

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by
mutations in the dystrophin gene that result in the absence of functional protein. Antisense …

The dysferlinopathies comprise a group of untreatable muscle disorders including limb
girdle muscular dystrophy type 2B, Miyoshi myopathy, distal anterior compartment …

The extracellular matrix (ECM) provides a solid scaffold and signals to cells through ECM
receptors. The cell–matrix interactions are crucial for normal biological processes and when …

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 …