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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Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Hindawi Publishing Corporation 2012
Témata:
Research Article
On-line přístup: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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