Antisense PMO cocktails effectively skip dystrophin exons 45-55 in myotubes transdifferentiated from DMD patient fibroblasts

Antisense-mediated exon skipping has made significant progress as a therapeutic platform in recent years, especially in the case of Duchenne muscular dystrophy (DMD). Despite FDA approval of eteplirsen–the first-ever antisense drug clinically marketed for DMD–exon skipping therapy still faces the si...

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Veröffentlicht in:PLoS One
Hauptverfasser: Lee, Joshua, Echigoya, Yusuke, Duddy, William, Saito, Takashi, Aoki, Yoshitsugu, Takeda, Shin’ichi, Yokota, Toshifumi
Format: Artigo
Sprache:Inglês
Veröffentlicht: Public Library of Science 2018
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Research Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5957359/
https://ncbi.nlm.nih.gov/pubmed/29771942
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0197084
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