The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Feb. 07, 2023
Filed:
May. 06, 2016
The Children's Medical Center Corporation, Boston, MA (US);
The Broad Institute, Inc., Cambridge, MA (US);
Massachusetts Institute of Technology, Cambridge, MA (US);
Daniel E. Bauer, Cambridge, MA (US);
Stuart H. Orkin, Brookline, MA (US);
Neville Sanjana, New York, NY (US);
Ophir Shalem, Albany, CA (US);
Feng Zhang, Cambridge, MA (US);
THE CHILDREN'S MEDICAL CENTER. CORPORATION, Boston, MA (US);
THE BROAD INSTITUTE, INC., Cambridge, MA (US);
MASSACHUSETTS INSTITUTE OF TECHNOLOGY, Cambridge, MA (US);
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.