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Article

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

10 Aug 2010

Abstract excerpt

Splice modulation using antisense oligonucleotides (AOs) has been shown to yield targeted exon exclusion to restore the open reading frame and generate truncated but partially functional dystrophin protein. This has been successfully demonstrated in dystrophin-deficient mdx mice and in Duchenne muscular dystrophy (DMD) patients. However, DMD is a systemic disease; successful therapeutic exploitation of this...

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PMID: 20700113
DOI: 10.1038/mt.2010.151

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