An exon skipping PPMO has demonstrated dramatic effects in the prevention and treatment of severely affected, dystrophin and utrophin-deficient mice, preventing severe deterioration of the treated animals and extending their lifespan. These findings were published online today in the journal Molecular Therapy and support the promise of this therapeutic approach for the treatment of Duchenne muscular dystrophy (DMD). These results were published by researchers at University of Oxford, AVI BioPharma, Inc. (Nasdaq: AVII) and the University of Western Australia, Perth. DMD is an incurable muscle–wasting disease associated with errors in the gene that makes dystrophin. Studies and research have shown that the ability to skip certain exons in dystrophin pre-mRNA could circumvent these dystrophin gene errors and provide a potential treatment for DMD patients. The paper "Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping" details the successful exon skipping and treatment of utrophin/dystrophin double knockout (dKO) mice with a cell-penetrating peptide-conjugated phosphorodiamidate morpholino oligomers (PPMO) targeting exon 23 in dystrophin pre-mRNA.
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