Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy

Cardiac failure is a major cause of mortality in patients with Duchenne muscular dystrophy (DMD). Antisense-mediated exon skipping has the ability to correct out-of-frame mutations in DMD to produce truncated but functional dystrophin. Traditional antisense approaches have however been limited by th...

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發表在:PLoS One
Main Authors: Blain, Alison M., Greally, Elizabeth, McClorey, Graham, Manzano, Raquel, Betts, Corinne A., Godfrey, Caroline, O’Donovan, Liz, Coursindel, Thibault, Gait, Mike J., Wood, Matthew J., MacGowan, Guy A., Straub, Volker W.
格式: Artigo
語言:Inglês
出版: Public Library of Science 2018
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Research Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6005479/
https://ncbi.nlm.nih.gov/pubmed/29912990
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0198897
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