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Brief Communication
Nature Medicine 12, 175 - 177 (2006)
Published online: 29 January 2006 | doi:10.1038/nm1345
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
Julia Alter1, Fang Lou2, Adam Rabinowitz1, HaiFang Yin1, Jeffrey Rosenfeld3, Steve D Wilton4, Terence A Partridge1 & Qi Long Lu3
Abstract
For the majority of Duchenne muscular dystrophy (DMD) mutations, antisense oligonucleotide (AON)-mediated exon skipping has the potential to restore a functional protein. Here we show that weekly intravenous injections of morpholino phosphorodiamidate (morpholino) AONs induce expression of functional levels of dystrophin in body-wide skeletal muscles of the dystrophic mdx mouse, with resulting improvement in muscle function. Although the level of dystrophin expression achieved varies considerably between muscles, antisense therapy may provide a realistic hope for the treatment of a majority of individuals with DMD.
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