Duchenne muscular dystrophy (DMD), the most frequently inherited muscle disease in childhood, is caused by mutations in the gene encoding dystrophin. The modification of dystrophin mRNA splicing, called exon-skipping, is a promising therapeutic strategy. This approach uses small molecules, anti-sense oligonucleotides, that “repair” the open reading frame of the Dmd gene through skipping of exons, which flank the original mutation and lead to the production of truncated dystrophin. Animal models play an indispensable role in the development and optimization of therapeutic approaches for DMD as well as in understanding the role dystrophin in the muscle.

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