Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice

Antisense therapy with both chemistries of phosphorodiamidate morpholino oligomers (PMOs) and 2′-O-methyl phosphorothioate has demonstrated the capability to induce dystrophin expression in Duchenne muscular dystrophy (DMD) patients in phase II–III clinical trials with benefit in muscle functions. H...

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Detalhes bibliográficos
Main Authors: Wu, B, Cloer, C, Lu, P, Milazi, S, Shaban, M, Shah, SN, Marston-Poe, L, Moulton, HM, Lu, QL
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4167372/
https://ncbi.nlm.nih.gov/pubmed/24942628
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2014.53
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