Oligomers
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United States of America Patent
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Jun 11, 2013
Issued Date -
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app pub date -
Nov 30, 2011
filing date -
Sep 11, 2008
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Abstract
Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.
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