Ideally, this would increase the quantity of functional dystrophin in affected DMD tissues to levels of therapeutic value. AOs may be introduced into myotubes to redirect the splicing of GRMD dystrophin pre-mRNA to restore the reading frame. Recently, genetic therapy with antisense oligonucleotides (AOs) has attracted special interest as a novel therapeutic approach for DMD. As a result, affected dogs have drastically reduced levels of dystrophin and its mRNA transcript. The GRMD mutation, a base change from A to G in the 3' splice acceptor site of intron 6, results in exon 7 skipping which disrupts the translational reading frame. Unlike the mdx model, the GRMD dog more accurately reflects the phenotype shown by human DMG patients, making the model better suited for the investigation and assessment of potential therapeutic approaches. Golden Retriever Muscular Dystrophy (GRMD), an animal model of DMD is a fatal degenerative myopathy. The development of an effective therapy for Duchenne Muscular Dystrophy (DMD) is one of the primary goals of all DMD/Becker Muscular Dystrophy (BMD) research.
