Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

BACKGROUND: We report clinical safety and biochemical efficacy from a dose-ranging study of intravenously administered AVI-4658 phosphorodiamidate morpholino oligomer (PMO) in patients with Duchenne muscular dystrophy. METHOD: We undertook an open-label, phase 2, dose-escalation study (0·5, 1·0, 2·0...

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Bibliografiset tiedot
Päätekijät: Cirak, Sebahattin, Arechavala-Gomeza, Virginia, Guglieri, Michela, Feng, Lucy, Torelli, Silvia, Anthony, Karen, Abbs, Stephen, Garralda, Maria Elena, Bourke, John, Wells, Dominic J, Dickson, George, Wood, Matthew JA, Wilton, Steve D, Straub, Volker, Kole, Ryszard, Shrewsbury, Stephen B, Sewry, Caroline, Morgan, Jennifer E, Bushby, Kate, Muntoni, Francesco
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Lancet Publishing Group 2011
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Articles
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC3156980/
https://ncbi.nlm.nih.gov/pubmed/21784508
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S0140-6736(11)60756-3
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