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.
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申请公布号 |
US2010168212(A1) |
申请公布日期 |
2010.07.01 |
申请号 |
US20090556626 |
申请日期 |
2009.09.10 |
申请人 |
ROYAL HOLLOWAY, UNIVERSITY OF LONDON |
发明人 |
POPPLEWELL LINDA;GRAHAM IAN;DICKSON JOHN GEORGE |
分类号 |
A61K31/7088;C07H21/04;C12N15/63 |
主分类号 |
A61K31/7088 |
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