| 发明名称 |
COMPOSITIONS AND METHODS TO TREATING HEMOGLOBINOPATHIES |
| 摘要 |
Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels. |
| 申请公布号 |
WO2015164750(A2) |
申请公布日期 |
2015.10.29 |
| 申请号 |
WO2015US27527 |
申请日期 |
2015.04.24 |
| 申请人 |
CHILDREN'S MEDICAL CENTER CORPORATION |
发明人 |
MILSOM, MICHAEL;WILLIAMS, DAVID A.;GREGORY, RICHARD |
| 分类号 |
A61K48/00 |
主分类号 |
A61K48/00 |
| 代理机构 |
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