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Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse.
Rimessi P, Sabatelli P, Fabris M, Braghetta P, Bassi E, Spitali P, Vattemi G, Tomelleri G, Mari L, Perrone D, Medici A, Neri M, Bovolenta M, Martoni E, Maraldi NM, Gualandi F, Merlini L, Ballestri M, Tondelli L, Sparnacci K, Bonaldo P, Caputo A, Laus M, Ferlini A. Rimessi P, et al. Among authors: perrone d. Mol Ther. 2009 May;17(5):820-7. doi: 10.1038/mt.2009.8. Epub 2009 Feb 24. Mol Ther. 2009. PMID: 19240694 Free PMC article.
Exon skipping-mediated dystrophin reading frame restoration for small mutations.
Spitali P, Rimessi P, Fabris M, Perrone D, Falzarano S, Bovolenta M, Trabanelli C, Mari L, Bassi E, Tuffery S, Gualandi F, Maraldi NM, Sabatelli-Giraud P, Medici A, Merlini L, Ferlini A. Spitali P, et al. Among authors: perrone d. Hum Mutat. 2009 Nov;30(11):1527-34. doi: 10.1002/humu.21092. Hum Mutat. 2009. PMID: 19760747
Persistent dystrophin protein restoration 90 days after a course of intraperitoneally administered naked 2'OMePS AON and ZM2 NP-AON complexes in mdx mice.
Bassi E, Falzarano S, Fabris M, Gualandi F, Merlini L, Vattemi G, Perrone D, Marchesi E, Sabatelli P, Sparnacci K, Laus M, Bonaldo P, Rimessi P, Braghetta P, Ferlini A. Bassi E, et al. Among authors: perrone d. J Biomed Biotechnol. 2012;2012:897076. doi: 10.1155/2012/897076. Epub 2012 Oct 2. J Biomed Biotechnol. 2012. PMID: 23091362 Free PMC article.
167 results