发明名称 Oligomers
摘要 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.
申请公布号 US8552172(B2) 申请公布日期 2013.10.08
申请号 US201113307926 申请日期 2011.11.30
申请人 POPPLEWELL LINDA;GRAHAM IAN;DICKSON JOHN GEORGE 发明人 POPPLEWELL LINDA;GRAHAM IAN;DICKSON JOHN GEORGE
分类号 C07H21/04 主分类号 C07H21/04
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