Long-term administration of antisense oligonucleotides into the paraspinal muscles of mdx mice reduces kyphosis.


The mdx mouse model of muscular dystrophy has a premature stop codon preventing production of dystrophin. This results in a progressive phenotype causing centronucleation of skeletal muscle fibers, muscle weakness, and fibrosis and kyphosis. Antisense oligonucleotides alter RNA splicing to exclude the nonsense mutation, while still maintaining the open… (More)
DOI: 10.1152/japplphysiol.00068.2008

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