| 发明名称 | Therapeutic compounds | ||
| 摘要 | The present invention is directed to RNA interference (RNAi) molecules targeted against a Huntington's disease nucleic acid sequence, and methods of using these RNAi molecules to treat Huntington's disease. | ||
| 申请公布号 | US9181544(B2) | 申请公布日期 | 2015.11.10 |
| 申请号 | US201213985023 | 申请日期 | 2012.02.13 |
| 申请人 | University of Iowa Research Foundation | 发明人 | Davidson Beverly L.;Boudreau Ryan L. |
| 分类号 | C12N15/11;C07H19/00;C12N15/113 | 主分类号 | C12N15/11 |
| 代理机构 | Viksnins Harris & Padys PLLP | 代理人 | Viksnins Harris & Padys PLLP |
| 主权项 | 1. A nucleic acid encoding an artificial primary miRNA transcript (pri-miRNA) consisting of, in order of position, a 5′-flanking region, a non-guide region, a loop region, a guide region, and a 3′-flanking region, wherein the guide region comprises a sequence 100% identical to cgaccaugcgagccagca (miHDS.1 guide. SEQ ID NO:7) and the non-guide region is at least 80% complementary to the guide region. | ||
| 地址 | Iowa City IA US | ||