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2006 | 1 |
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Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human.
Neuromuscul Disord. 2007 Dec;17(11-12):913-8. doi: 10.1016/j.nmd.2007.07.005. Epub 2007 Sep 7.
Neuromuscul Disord. 2007.
PMID: 17826093
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.
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.
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Intronic breakpoint definition and transcription analysis in DMD/BMD patients with deletion/duplication at the 5' mutation hot spot of the dystrophin gene.
Gualandi F, Rimessi P, Trabanelli C, Spitali P, Neri M, Patarnello T, Angelini C, Yau SC, Abbs S, Muntoni F, Calzolari E, Ferlini A.
Gualandi F, et al.
Gene. 2006 Mar 29;370:26-33. doi: 10.1016/j.gene.2005.11.002. Epub 2006 Jan 24.
Gene. 2006.
PMID: 16439068
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Antisense modulation of both exonic and intronic splicing motifs induces skipping of a DMD pseudo-exon responsible for x-linked dilated cardiomyopathy.
Rimessi P, Fabris M, Bovolenta M, Bassi E, Falzarano S, Gualandi F, Rapezzi C, Coccolo F, Perrone D, Medici A, Ferlini A.
Rimessi P, et al.
Hum Gene Ther. 2010 Sep;21(9):1137-46. doi: 10.1089/hum.2010.010.
Hum Gene Ther. 2010.
PMID: 20486769
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