Summary and Future Direction

Antisense oligomer-mediated exon skipping has now been demonstrated as one of the most promising experimental therapies for DMD patients. With the most potent chemistry in conjunction with the application of delivery-enabling polymers, the efficacy of the therapy systemically has now been well-established in models of both dystrophic mice and dogs. While the long-term efficacy of the therapy to DMD boys remains to be established, successful restoration of dystrophin expression in both cardiac and skeletal muscles provides hope that the therapy can be effective to rescue dystrophic phenotypes of both skeletal and cardiac muscle. As clinical trials of systemic treatment with highly effective antisense oligomers are well under way, this therapy could represent the first realization of gene therapy to muscular dystrophies.

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