| 发明名称 |
MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES |
| 摘要 |
The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies. |
| 申请公布号 |
EP2334794(A2) |
申请公布日期 |
2011.06.22 |
| 申请号 |
EP20090813730 |
申请日期 |
2009.09.14 |
| 申请人 |
THE CHILDREN'S MEDICAL CENTER CORPORATION;PRESIDENT AND FELLOWS OF HARVARD COLLEGE |
发明人 |
ORKIN, STUART, H.;SANKARAN, VIJAY, G. |
| 分类号 |
C12N15/63;A61K31/7088;C12N15/113 |
主分类号 |
C12N15/63 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
|
| 地址 |
|
