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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2018
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6005479/
https://www.ncbi.nlm.nih.gov/pubmed/29912990
http://dx.doi.org/10.1371/journal.pone.0198897

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