发明授权
- 专利标题: Oligomers
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申请号: US14736154申请日: 2015-08-24
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公开(公告)号: US09650632B2公开(公告)日: 2017-05-16
- 发明人: Linda Popplewell , Ian Graham , John George Dickson
- 申请人: Royal Holloway, University of London
- 申请人地址: GB Egham, Surrey
- 专利权人: Royal Holloway, University of London
- 当前专利权人: Royal Holloway, University of London
- 当前专利权人地址: GB Egham, Surrey
- 代理机构: Choate, Hall & Stewart LLP
- 主分类号: A61K48/00
- IPC分类号: A61K48/00 ; C12N15/113 ; A61K31/7088 ; C12N15/115
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.
公开/授权文献
- US20160024500A1 OLIGOMERS 公开/授权日:2016-01-28
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