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Persistent Dystrophin Protein Restoration 90 Days after a Course of Intraperitoneally Administered Naked 2′OMePS AON and ZM2 NP-AON Complexes in mdx Mice

In Duchenne muscular dystrophy, the exon-skipping approach has obtained proof of concept in animal models, myogenic cell cultures, and following local and systemic administration in Duchenne patients. Indeed, we have previously demonstrated that low doses (7.5 mg/Kg/week) of 2′-O-methyl-phosphorothi...

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書誌詳細
主要な著者: Bassi, Elena, Falzarano, Sofia, Fabris, Marina, Gualandi, Francesca, Merlini, Luciano, Vattemi, Gaetano, Perrone, Daniela, Marchesi, Elena, Sabatelli, Patrizia, Sparnacci, Katia, Laus, Michele, Bonaldo, Paolo, Rimessi, Paola, Braghetta, Paola, Ferlini, Alessandra
フォーマット: Artigo
言語:Inglês
出版事項: Hindawi Publishing Corporation 2012
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC3471065/
https://ncbi.nlm.nih.gov/pubmed/23091362
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1155/2012/897076
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