| 发明名称 |
TARGETING BCL11A DISTAL REGULATORY ELEMENTS FOR FETAL HEMOGLOBIN REINDUCTION |
| 摘要 |
Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels. |
| 申请公布号 |
EP2925864(A4) |
申请公布日期 |
2016.07.13 |
| 申请号 |
EP20130858006 |
申请日期 |
2013.11.27 |
| 申请人 |
CHILDREN'S MEDICAL CENTER CORPORATION |
发明人 |
ORKIN, STUART H.;BAUER, DANIEL E.;XU, JIAN |
| 分类号 |
C12N5/10;A61K35/12;A61K38/46;C07K14/47;C07K14/805;C12N5/0789;C12N9/22;C12N15/01;C12N15/09;C12N15/10;C12N15/63 |
主分类号 |
C12N5/10 |
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