Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD

Abstract

Manipulation of pre-mRNA splicing by antisense oligonucleotides (AOs) offers considerable potential for a number of genetic disorders. One of these is Duchenne muscular dystrophy (DMD), where mutations in the dystrophin gene typically result in premature termination of translation that causes a loss of functional protein. AOs can induce exon skipping such… (More)
DOI: 10.1038/sj.gt.3302800

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Cite this paper

@article{Mcclorey2006AntisenseOE, title={Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD}, author={Graham Mcclorey and Hong M. Moulton and P L Iversen and S. Fletcher and S D Wilton}, journal={Gene Therapy}, year={2006}, volume={13}, pages={1373-1381} }