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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Bibliografische gegevens
Hoofdauteurs:	Alison M Blain, Elizabeth Greally, Graham McClorey, Raquel Manzano, Corinne A Betts, Caroline Godfrey, Liz O'Donovan, Thibault Coursindel, Mike J Gait, Matthew J Wood, Guy A MacGowan, Volker W Straub
Formaat:	Artikel
Taal:	English
Gepubliceerd in:	Public Library of Science (PLoS) 2018-01-01
Reeks:	PLoS ONE
Online toegang:	http://europepmc.org/articles/PMC6005479?pdf=render

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http://europepmc.org/articles/PMC6005479?pdf=render

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

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