Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction

US11572543B2 · kind B2 · utility

Assignees

• Children's Medical Center Corporation · US
• The Broad Institute, Inc. · US
• Massachusetts Institute of Technology · US

Inventors

• Daniel E. Bauer · Cambridge, US
• Stuart H. Orkin · Brookline, US
• Neville Espi Sanjana · New York, US
• Ophir Shalem · Albany, US
• Feng Zhang · Cambridge, US

Key dates

• Filing date: May 6, 2016
• Grant date: Feb 7, 2023
• Expiry date: Oct 13, 2036

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2740/15041
• WIPO field: Biotechnology
• WIPO sector: Chemistry

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.