公开(公告)号：EP3763814A1

公开(公告)日：2021-01-13

申请号：EP20185077.3

申请日：2016-05-06

申请人： The Children's Medical Center Corporation ,  The Broad Institute, Inc. ,  Massachusetts Institute of Technology

发明人： BAUER, Daniel E. ,  ORKIN, Stuart H. ,  SANJANA, Neville ,  SHALEM, Ophir ,  ZHANG, Feng

IPC分类号： C12N15/10 ,  C12N5/078 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

摘要： Provided herein are nucleic acid molecules that target the BCL11A enhancer functional regions, compositions comprising the nucleic acid molecules and methods 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. In particular, the nucleic acid molecules target the +62, +58, and/or the +55 enhancer functional regions.

公开(公告)号：EP3294873B1

公开(公告)日：2020-08-19

申请号：EP16793259.9

申请日：2016-05-06

申请人： The Children's Medical Center Corporation ,  The Broad Institute, Inc. ,  Massachusetts Institute of Technology

发明人： BAUER, Daniel E. ,  ORKIN, Stuart H. ,  SANJANA, Neville ,  SHALEM, Ophir ,  ZHANG, Feng

IPC分类号： C12N15/10 ,  C12N5/078 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86