Rescue of dystrophin mRNA of Duchenne muscular dystrophy by inducing exon skipping.
Duchenne muscular dystrophy (DMD) is a fatal muscle-wasting disease, and its victims usually succumb in their twenties. Many studies, including investigations into gene-replacement therapy, have been conducted in a search for a treatment for DMD, and the most promising treatment to date is rescue of mutant dystrophin mRNA by induction of exon skipping. On the basis of results from the molecular analysis of dystrophin Kobe, we propose a treatment for DMD in which antisense oligonucleotides induce exon skipping to edit out-of-frame dystrophin mRNA into in-frame, thereby converting severe DMD to a milder form. Here we review the progress of development of this alternative treatment, with a special focus on dystrophin Kobe.
Department of Pediatrics, Kobe University Graduate School of Medicine, Chuo, Japan. matsuo@kobe-u.ac.jp
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Matsuo
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Takeshima
D016189:Dystrophin / Q000235:genetics*; D005091:Exons; D017353:Gene Deletion; D018390:Gene Targeting; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics*; D020319:Oligodeoxyribonucleotides, Antisense / Q000235:genetics* / Q000494:pharmacology; D012326:RNA Splicing
D016189:Dystrophin; D020319:Oligodeoxyribonucleotides, Antisense