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Article

Elusive sources of variability of dystrophin rescue by exon skipping

1 Dec 2015

Abstract excerpt

BACKGROUND: Systemic delivery of anti-sense oligonucleotides to Duchenne muscular dystrophy (DMD) patients to induce de novo dystrophin protein expression in muscle (exon skipping) is a promising therapy. Treatment with Phosphorodiamidate morpholino oligomers (PMO) lead to shorter de novo dystrophin protein in both animal models and DMD boys who otherwise lack dystrophin; however, restoration of dystrophin has...

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PMID: 26634117
DOI: 10.1186/s13395-015-0070-6

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