- 专利标题： RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF MYOTILIN
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申请号： EP12774899.4申请日： 2012-04-20
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公开(公告)号： EP2699673B1公开(公告)日： 2018-09-19
- 发明人: HARPER, Scott Quenton , LIU, Jian
- 申请人： Nationwide Children's Hospital, Inc.
- 申请人地址： 700 Children's Drive Room W172 Columbus, OH 43205 US
- 专利权人： Nationwide Children's Hospital, Inc.
- 当前专利权人： Nationwide Children's Hospital, Inc.
- 当前专利权人地址： 700 Children's Drive Room W172 Columbus, OH 43205 US
- 代理机构： Walker, Ross Thomson
- 优先权： US201161478012P 20110421
- 国际公布： WO2012145597 20121026
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; A61K48/00 ; C12N15/864 ; A61K31/7088 ; C07K14/47 ; C12N7/01
摘要：
The present invention relates to RNA interference-based methods for inhibiting the expression of the myotilin gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of myotilin. The methods have application in the treatment of muscular dystrophies such as Limb Girdle Muscular Dystrophy Type 1A.
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