Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice

BACKGROUND: Exon skipping oligonucleotides (ESOs) of 2'O-Methyl (2'OMe) and morpholino chemistry have been shown to restore dystrophin expression in muscle fibers from the mdx mouse, and are currently being tested in phase I clinical trials for Duchenne Muscular Dystrophy (DMD). However, E...

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Autori principali: Williams, Jason H, Schray, Rebecca C, Sirsi, Shashank R, Lutz, Gordon J
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2008
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2362111/
https://ncbi.nlm.nih.gov/pubmed/18384691
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1472-6750-8-35
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