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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...
Autores principales: | 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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Hindawi Publishing Corporation
2012
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3471065/ https://www.ncbi.nlm.nih.gov/pubmed/23091362 http://dx.doi.org/10.1155/2012/897076 |
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