We report the restoration of the 430-kD dystrophin in mdx, the mouse model of Duchenne
muscular dystrophy, by expression of a single-copy recombinant dystrophin transgene …

Advances in molecular genetics are being applied to several of the inherited muscular
dystrophies. Strategies designed to identify DNA polymorphisms that are closely linked to …

Recent progress in molecular genetics has resulted in more insight in the molecular biology
of muscles and the muscular dystrophies, and has provided more reliable tools for prenatal …

The muscular dystrophies are a clinically and genetically heterogeneous group of skeletal
muscle-wasting diseases that differ widely in their frequency and pattern of cardiac …

N hereditary muscular dystrophy (symbol dy) in the mouse (MICHELSON, AR~~~~~~ and
HARMAN 1955) appeared as, a deviant in the strain 129/Re, which is highly inbred. All …

Duchenne muscular dystrophy (DMD) is a degenerative disorder of the skeletal muscle in
human and is caused by mutations in the dystrophin gene. Themdxmouse is a spontaneous …

Successful gene therapy of Duchenne muscular dystrophy may require the lifelong
expression of a therapeutic gene in all affected muscles. The most promising gene delivery …

The histopathological aspects of leg muscles in young, adult and old mdx mice were
compared. Before 1 yr of age, the mutant showed a vigorous regeneration, following the …

Clinical trials represent a critical avenue for new treatment development, where early
phases (I, I/II) are designed to test safety and effectiveness of new therapeutics or diagnostic …

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