Duchenne muscular dystrophy (DMD) is a severe genetic disease caused by different mutations in dystrophin gene. One of the promising treatment strategies which was already approved by Food and Drug Administration is exon-skipping. The approach is based on reading frame restoration after one or more exons exclusion from mRNA splicing. It is driven by specific antisense oligonucleotides (AON). We created new murine DMD model with deletion of exon from 8 to 34 and used it for exon-skipping approach test.

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