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Article

Toward an Oligonucleotide Therapy for Duchenne Muscular Dystrophy: A Complex Development Challenge

31 Mar 2010

Abstract excerpt

Antisense oligonucleotide (AO)-mediated exon skipping is a promising new therapy for Duchenne muscular dystrophy (DMD), recently demonstrating proof of principle for restoring the absent dystrophin protein in DMD patients. However, the range of AO chemistries available for exon skipping is limited; effective systemic dystrophin protein restoration has yet to be demonstrated and will be required for disease...

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PMID: 20424011
DOI: 10.1126/scitranslmed.3000512

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