Article
Evaluation of exon-skipping strategies for Duchenne muscular dystrophy utilizing dystrophin-deficient zebrafish.
Journal of cellular and molecular medicine - 1 Dec 2011
Berger Joachim, Berger Silke, Jacoby Arie S, Wilton Steve D, Currie Peter D
Abstract excerpt
Duchenne muscular dystophy (DMD) is a severe muscle wasting disease caused by mutations in the dystrophin gene. By utilizing antisense oligonucleotides, splicing of the dystrophin transcript can be altered so that exons harbouring a mutation are excluded from the mature mRNA. Although this approach has been shown to be effective to restore partially functional dystrophin protein, the level of dystrophin protein...
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