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        Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

        Author
        Alter, Julia
        Lou, Fang
        Rabinowitz, Adam
        Yin, HaiFang
        Rosenfeld, Jeffrey
        Wilton, Steve D
        Partridge, Terence A
        Lu, Qi Long
        Attention
        2299/6502
        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.
        Publication date
        2006
        Published in
        Nature Medicine
        Published version
        https://doi.org/10.1038/nm1345
        Other links
        http://hdl.handle.net/2299/6502
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