- 专利标题： TARGETING BCL11A DISTAL REGULATORY ELEMENTS FOR FETAL HEMOGLOBIN REINDUCTION
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申请号： EP18203436.3申请日： 2013-11-27
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公开(公告)号： EP3502240A1公开(公告)日： 2019-06-26
- 发明人: ORKIN, Stuart H. , BAUER, Daniel E. , XU, Jian
- 申请人： The Children's Medical Center Corporation
- 申请人地址： 55 Shattuck Street Boston, Massachusetts 02115 US
- 专利权人： The Children's Medical Center Corporation
- 当前专利权人： The Children's Medical Center Corporation
- 当前专利权人地址： 55 Shattuck Street Boston, Massachusetts 02115 US
- 代理机构： Kehoe, Laura Ellen
- 优先权： US201261730323P 20121127; US201261730369P 20121127; US201361776144P 20130311; US201361889174P 20131010
- 主分类号： C12N5/10
- IPC分类号： C12N5/10 ; C12N5/0789 ; C12N15/09 ; A61K35/12 ; A61K38/46 ; C07K14/805 ; C12N15/10 ; C12N15/63 ; C07K14/47 ; C12N15/01 ; C12N9/22
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.
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