- 专利标题： Methods and agents to increase therapeutic dystrophin expression in muscle
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申请号： US14249748申请日： 2014-04-10
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公开(公告)号： US09637738B2公开(公告)日： 2017-05-02
- 发明人: Eric Hoffman , Alyson Fiorillo
- 申请人： Eric Hoffman , Alyson Fiorillo
- 申请人地址： US MD Rockville
- 专利权人： Reveragen Biopharma, Inc.
- 当前专利权人： Reveragen Biopharma, Inc.
- 当前专利权人地址： US MD Rockville
- 代理商 Dennis A. Bennett
- 主分类号： C12N15/11
- IPC分类号： C12N15/11 ; C12N15/113 ; A61K31/573 ; A61K31/7088 ; A61K45/06
摘要：
Agents and methods for increasing dystrophin protein expression in muscle through blocking of specific microRNAs and microRNA binding sites on the dystrophin 3′ untranslated region (miR-146a, miRNA-146b, miR-223, miR-320a, miR374a, and miR-382). Methods for increasing the amount of dystrophin useful for effective therapeutic intervention for Becker muscular dystrophy, Duchenne muscular dystrophy, and other disorders where loss of dystrophin from muscle causes pathology.
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