Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

We previously conducted a proof of principle; dose escalation study in Duchenne muscular dystrophy (DMD) patients using the morpholino splice-switching oligonucleotide AVI-4658 (eteplirsen) that induces skipping of dystrophin exon 51 in patients with relevant deletions, restores the open reading fra...

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Autori principali: Cirak, Sebahattin, Feng, Lucy, Anthony, Karen, Arechavala-Gomeza, Virginia, Torelli, Silvia, Sewry, Caroline, Morgan, Jennifer E, Muntoni, Francesco
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2012
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3277241/
https://ncbi.nlm.nih.gov/pubmed/22086232
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.248
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