Article
Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides.
Neuromuscular disorders : NMD - 1 Jul 1999
Wilton S D, Lloyd F, Carville K, Fletcher S, Honeyman K, Agrawal S, Kole R
Abstract excerpt
The mdx mouse, which carries a nonsense mutation in exon 23 of the dystrophin gene, has been used as an animal model of Duchenne muscular dystrophy to evaluate cell or gene replacement therapies. Despite the mdx mutation, which should preclude the synthesis of a functional dystrophin protein, rare, naturally occurring dystrophin-positive fibres have been observed in mdx muscle tissue. These dystrophin-positive...
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