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Muscular Dystrophy
Chimeraplast-mediated exon skipping restores muscle cell dystrophin expression
July 24th, 2003
Chimeraplast-mediated exon skipping restores muscle cell dystrophin expression. According to recent research from the United States, "The most common types of dystrophin gene mutations that cause Duchenne muscular dystrophy (DMD) are large deletions that result in a shift of the translational reading frame. Such mutations generally lead to a complete absence of dystrophin protein in the muscle cells of affected individuals. Any therapeutic modality that could restore the reading frame would have the potential to substantially reduce the severity of the disease by allowing the production of an internally deleted, but partially functional, dystrophin protein as occurs in...
Source: Gene Therapy Weekly (2003-07-24)
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