公开(公告)号：EP3702459A1

公开(公告)日：2020-09-02

申请号：EP18870546.1

申请日：2018-10-26

申请人： Edigene Inc.

发明人： YUAN, Pengfei ,  FANG, Riguo ,  YU, Lingling ,  XIA, Penghui ,  WANG, Jia ,  LIANG, Fucai

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(公告)号：EP3702458A1

公开(公告)日：2020-09-02

申请号：EP18870256.7

申请日：2018-10-26

申请人： Edigene Inc.

发明人： YUAN, Pengfei ,  FANG, Riguo ,  YU, Lingling ,  XIA, Penghui ,  WANG, Jia ,  LIANG, Fucai

IPC分类号： C12N15/113 ,  C12N5/22 ,  C12N9/22 ,  A61K35/18 ,  A61P7/06

摘要： Provided is a method for gene editing of an enhancer site of the BCL11A in hematopoietic stem cells. The genetically modified hematopoietic stem cells have the functions of normal cells, and can significantly increase the expression of fetal hemoglobin so as to be used in the treatment of β thalassemia and sickle cell anemia.