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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Main Authors: 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
Format: Artigo
Jezik:Inglês
Izdano: Hindawi Publishing Corporation 2012
Teme:
Research Article
Online dostop: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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