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- W2013234807 abstract "Mutations in the dystrophin gene that create frame-shifts or premature stop codons on the mRNA are responsible of the severe Duchenne Muscular Dystrophy (DMD), while mutations that preserve the reading frame result in either milder or asymptomatic phenotypes. The exon skipping stratagem makes use of compounds designed to target key motifs on pre-mRNA to re-direct splicing in order to eliminate detrimental exons and rescue the production of shorter-proteins still functional. We have achieved persistent |[ldquo]|on demand alternative splicing|[rdquo]| that removes several consecutive exons on the dystrophin mRNA of the GRMD dog, by single administration of AAV vectors expressing antisense sequences coupled to a modified U7 short nuclear (sn) RNA. We report the sustained production of dystrophin at physiological levels in entire groups of muscles at the scale of a large size animal model of DMD." @default.
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- W2013234807 date "2006-01-01" @default.
- W2013234807 modified "2023-10-18" @default.
- W2013234807 title "918. Rescue of Dystrophin in the GRMD Dog by Multi-Exon Skipping Using Engineered U7 snRNAs" @default.
- W2013234807 doi "https://doi.org/10.1016/j.ymthe.2006.08.1008" @default.
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