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Article

Efficacy of systemic morpholino exon‐skipping in duchenne dystrophy dogs

13 Mar 2009

Abstract excerpt

OBJECTIVE: Duchenne muscular dystrophy (DMD) is caused by the inability to produce dystrophin protein at the myofiber membrane. A method to rescue dystrophin production by antisense oligonucleotides, termed exon-skipping, has been reported for the mdx mouse and in four DMD patients by local intramuscular injection. We sought to test efficacy and toxicity of intravenous oligonucleotide (morpholino)-induced exon...

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PMID: 19288467
DOI: 10.1002/ana.21627

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