- 专利标题： METHOD FOR IMPROVING FETAL HEMOGLOBIN EXPRESSION
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申请号： EP18870546.1申请日： 2018-10-26
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公开(公告)号： EP3702459A1公开(公告)日： 2020-09-02
- 发明人: YUAN, Pengfei , FANG, Riguo , YU, Lingling , XIA, Penghui , WANG, Jia , LIANG, Fucai
- 申请人： Edigene Inc.
- 申请人地址： Life Science Park Floor 2, Building 2 22 KeXueYuan Road Changping District Beijing 102206 CN
- 专利权人： Edigene Inc.
- 当前专利权人： Edigene Inc.
- 当前专利权人地址： Life Science Park Floor 2, Building 2 22 KeXueYuan Road Changping District Beijing 102206 CN
- 代理机构： Lavoix
- 优先权： CN201711027708 20171027
- 国际公布： WO2019080917 20190502
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; C12N5/22 ; C12N9/22 ; A61K35/18 ; A61P7/06
摘要：
Provided is a method for genetically editing an enhancer locus of hematopoietic stem cell BCL11A, comprising: disrupting the CTTCCT region of a BCL11A genome by gene editing technology. The genetically edited hematopoietic stem cells have normal cell functions and can significantly increase the expression of fetal hemoglobin, thereby being used for the treatment of β thalassemia and sickle cell anemia.
公开/授权文献
- EP3702459B1 METHOD FOR IMPROVING FETAL HEMOGLOBIN EXPRESSION 公开/授权日：2023-10-04
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