发明申请
WO2017147467A1 RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INDUCING DUX4 EXON SKIPPING
审中-公开
重组病毒产品和诱导DUX4 EXON跳跃的方法
- 专利标题： RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INDUCING DUX4 EXON SKIPPING
- 专利标题（中）： 重组病毒产品和诱导DUX4 EXON跳跃的方法
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申请号： PCT/US2017/019422申请日： 2017-02-24
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公开(公告)号： WO2017147467A1公开(公告)日： 2017-08-31
- 发明人: HARPER, Scott, Quenton
- 申请人： RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
- 申请人地址： 700 Children's Drive Room W172 Columbus, OH 43205 US
- 专利权人： RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
- 当前专利权人： RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
- 当前专利权人地址： 700 Children's Drive Room W172 Columbus, OH 43205 US
- 代理机构： NOLAND, Greta, E. et al.
- 优先权： US62/300,500 20160226
- 主分类号： C12Q1/68
- IPC分类号： C12Q1/68 ; C12N15/09 ; C12N5/10 ; C12N15/11 ; C12N15/113
摘要：
The present invention relates to methods for shifting the splicing profile of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding U7-based small nuclear RNAs to induce DUX4 exon- skipping and the expression of shortened forms of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.
