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Article

Therapeutic exon skipping for dysferlinopathies?

10 Feb 2010

Abstract excerpt

Antisense-mediated exon skipping is a promising therapeutic approach for Duchenne muscular dystrophy (DMD) currently tested in clinical trials. The aim is to reframe dystrophin transcripts using antisense oligonucleotides (AONs). These hide an exon from the splicing machinery to induce exon skipping, restoration of the reading frame and generation of internally deleted, but partially functional proteins. It thus...

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PMID: 20145676
DOI: 10.1038/ejhg.2010.4

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