Article
Redirecting splicing to address dystrophin mutations: molecular by-pass surgery.
Progress in molecular and subcellular biology - 1 Jan 2006
Wilton Stephen D, Fletcher Susan
Abstract excerpt
Mutations in the dystrophin gene that prevent synthesis of a functional protein lead to Duchenne muscular dystrophy (DMD), the most common serious childhood muscular dystrophy. The major isoform is produced in skeletal muscle and the size of the dystrophin gene and complexity of expression have posed great challenges to the development of a therapy for DMD. Considerable progress has been made in the areas of gene...
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