| 发明名称 |
AAV-BASED TREATMENT OF CHOLESTEROL-RELATED DISORDERS |
| 摘要 |
The invention in some aspects relates to methods and compositions for assessing the effectiveness of miRNA inhibitors. In other aspects of the invention, methods and compositions for treating cholesterol related disorders are provided. In one aspect of the invention, miRNA inhibitors against miR-122 and rAAV-based compositions comprising the same are provided. |
| 申请公布号 |
US2016208257(A1) |
申请公布日期 |
2016.07.21 |
| 申请号 |
US201614995699 |
申请日期 |
2016.01.14 |
| 申请人 |
University of Massachusetts |
发明人 |
Gao Guangping;Zamore Phillip D.;Xie Jun |
| 分类号 |
C12N15/113;C12N15/86;C12Q1/68;C12N7/00 |
主分类号 |
C12N15/113 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A method for treating a high cholesterol-related disorder in a subject, the method comprising:
administering an effective amount of a recombinant Adeno-Associated Virus (rAAV) to the subject, wherein the rAAV comprises at least one transgene that expresses a miRNA inhibitor that inhibits the expression of miR-122 in the subject. |
| 地址 |
Boston MA US |
