| 发明名称 |
HUNTINGTON'S DISEASE 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 |
US2016060624(A1) |
申请公布日期 |
2016.03.03 |
| 申请号 |
US201514717795 |
申请日期 |
2015.05.20 |
| 申请人 |
University of Iowa Research Foundation |
发明人 |
Davidson Beverly L.;Mas Monteys Alejandro |
| 分类号 |
C12N15/113;A61K9/00;A61K31/7088 |
主分类号 |
C12N15/113 |
| 代理机构 |
|
代理人 |
|
| 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 consists of SEQ ID NO: 37 (miHDss3), SEQ ID NO:6 (miHDS1v5U) or SEQ ID NO:7 (miHDS1v6A), and the non-guide region is at least 80% complementary to the guide region. |
| 地址 |
Iowa City IA US |
