Morpholino Oligomer-Induced Dystrophin Isoforms to Map the Functional Domains in the Dystrophin Protein

Abstract

Antisense oligonucleotide-mediated exon skipping is used to induce different dystrophin isoforms, one isoform missing amino acids encoded by DMD exons 56+57 and the other missing exons 58+59. Initial studies indicate that the latter is less functional, suggesting that mutations involving these exons may not respond to an exon skipping therapy.