发明公开
EP2699673A4 RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF MYOTILIN
有权
REKOMBINANTE VIRUSPRODUKTE UND VERFAHREN ZUR EXPRESSIONSHEMMUNG VON MYOTILIN
- 专利标题： RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF MYOTILIN
- 专利标题（中）： REKOMBINANTE VIRUSPRODUKTE UND VERFAHREN ZUR EXPRESSIONSHEMMUNG VON MYOTILIN
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申请号： EP12774899申请日： 2012-04-20
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公开(公告)号： EP2699673A4公开(公告)日： 2015-01-07
- 发明人: HARPER SCOTT QUENTON , LIU JIAN
- 申请人： NATIONWIDE CHILDRENS HOSPITAL
- 专利权人： NATIONWIDE CHILDRENS HOSPITAL
- 当前专利权人： NATIONWIDE CHILDRENS HOSPITAL
- 优先权： US201161478012 2011-04-21
- 主分类号： C12N7/01
- IPC分类号： C12N7/01 ; A61K31/7088 ; A61K48/00 ; C07K14/47 ; C12N15/86
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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