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

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...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Lu-Nguyen, Ngoc B, Jarmin, Susan A, Saleh, Amer F, Popplewell, Linda, Gait, Michael J, Dickson, George
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2015
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4579549/
https://ncbi.nlm.nih.gov/pubmed/25959011
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.88
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