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 US2013289096(A1) 申请公布日期 2013.10.31
申请号 US201313909132 申请日期 2013.06.04
申请人 ROYAL HOLLOWAY, UNIVERSITY OF LONDON 发明人 POPPLEWELL LINDA;GRAHAM IAN;DICKSON JOHN GEORGE
分类号 C12N15/113 主分类号 C12N15/113
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