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Article

Exon skipping: a first in class strategy for Duchenne muscular dystrophy

19 Dec 2016

Abstract excerpt

INTRODUCTION: Exon skipping is a therapeutic approach for Duchenne muscular dystrophy (DMD) that has been in development for close to two decades. This approach uses antisense oligonucleotides (AONs) to modulate pre-mRNA splicing of dystrophin transcripts to restore the disrupted DMD reading frame. The approach has moved from in vitro proof of concept studies to the clinical trial phase and marketing...

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PMID: 27936976
DOI: 10.1080/14712598.2017.1271872

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