Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.

Abstract

The fatal X-linked Duchenne muscular dystrophy (DMD), characterized by progressive muscle wasting and muscle weakness, is caused by mutations within the DMD gene. The use of antisense oligonucleotides (AOs) modulating pre-mRNA splicing to restore the disrupted dystrophin reading frame, subsequently generating a shortened but functional protein has emerged… (More)
DOI: 10.1038/mt.2015.88

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Cite this paper

@article{LuNguyen2015CombinationAT, title={Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice.}, author={Ngoc B Lu-Nguyen and Susan A Jarmin and Amer F. Alhaj Saleh and Linda J Popplewell and Michael J. Gait and George Dickson}, journal={Molecular therapy : the journal of the American Society of Gene Therapy}, year={2015}, volume={23 8}, pages={1341-1348} }