发明申请
US20170029849A1 RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF DUX4
审中-公开
重组病毒产品和抑制DUX4表达的方法
- 专利标题： RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF DUX4
- 专利标题（中）： 重组病毒产品和抑制DUX4表达的方法
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申请号： US15295591申请日： 2016-10-17
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公开(公告)号： US20170029849A1公开(公告)日： 2017-02-02
- 发明人: Scott Quenton Harper , Jian Liu , Sara Coppens , Lindsay Wallace
- 申请人： NATIONWIDE CHILDREN'S HOSPITAL, INC.
- 主分类号： C12N15/86
- IPC分类号： C12N15/86 ; C12N15/113
摘要：
The present invention relates to RNA interference-based methods for inhibiting the expression of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.
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