Invention Grant
- Patent Title: Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction
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Application No.: US15572523Application Date: 2016-05-06
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Publication No.: US11572543B2Publication Date: 2023-02-07
- Inventor: Daniel E. Bauer , Stuart H. Orkin , Neville Sanjana , Ophir Shalem , Feng Zhang
- Applicant: The Children's Medical Center Corporation , The Broad Institute, Inc. , Massachusetts Institute of Technology
- Applicant Address: US MA Boston; US MA Cambridge; US MA Cambridge
- Assignee: The Children's Medical Center Corporation,The Broad Institute, Inc.,Massachusetts Institute of Technology
- Current Assignee: The Children's Medical Center Corporation,The Broad Institute, Inc.,Massachusetts Institute of Technology
- Current Assignee Address: US MA Boston; US MA Cambridge; US MA Cambridge
- Agency: Nixon Peabody LLP
- Agent David S. Resnick; Jeanne Jodoin
- International Application: PCT/US2016/031224 WO 20160506
- International Announcement: WO2016/182917 WO 20161117
- Main IPC: C12N5/078
- IPC: C12N5/078 ; C12N15/10 ; C12N9/22 ; C12N15/11 ; C12N15/86

Abstract:
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.
Public/Granted literature
- US20180171297A1 TARGETING BCL11A ENHANCER FUNCTIONAL REGIONS FOR FETAL HEMOGLOBIN REINDUCTION Public/Granted day:2018-06-21
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