Cationic PMMA Nanoparticles Bind and Deliver Antisense Oligoribonucleotides Allowing Restoration of Dystrophin Expression in the mdx Mouse

For subsets of Duchenne muscular dystrophy (DMD) mutations, antisense oligoribonucleotide (AON)-mediated exon skipping has proven to be efficacious in restoring the expression of dystrophin protein. In the mdx murine model systemic delivery of AON, recognizing the splice donor of dystrophin exon 23,...

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Main Authors: Rimessi, Paola, Sabatelli, Patrizia, Fabris, Marina, Braghetta, Paola, Bassi, Elena, Spitali, Pietro, Vattemi, Gaetano, Tomelleri, Giuliano, Mari, Lara, Perrone, Daniela, Medici, Alessandro, Neri, Marcella, Bovolenta, Matteo, Martoni, Elena, Maraldi, Nadir M., Gualandi, Francesca, Merlini, Luciano, Ballestri, Marco, Tondelli, Luisa, Sparnacci, Katia, Bonaldo, Paolo, Caputo, Antonella, Laus, Michele, Ferlini, Alessandra
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2009
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Original Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835127/
https://ncbi.nlm.nih.gov/pubmed/19240694
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.8
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