ABSTRACT
Muscular dystrophies are a heterogeneous group of genetic disorders characterized by progressive muscle weakness and wasting. The most common and severe form of muscular dystrophy is Duchenne muscular dystrophy (DMD). DMD is an X-linked recessive disease characterized by a mutation in the gene encoding dystrophin, a myofiber stabilizing protein (1-3). A point mutation in exon 23 of the dystrophin gene causes a milder muscle wasting in mice (mdx) than that observed in humans (4). Dystrophin is a cytoskeletal protein mainly expressed in skeletal, cardiac, and smooth muscle, and in the brain, and is severely reduced or absent in DMD patients and mdx mice (4-7).
