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US 2019/0169615 A1Published application
Modulation of bcl11a for treatment of hemoglobinopathies
AssigneesChildren's Medical Center Corporation, President and Fellows of Harvard College
InventorsStuart H. ORKIN, Vijay G. SANKARAN
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Abstract
The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.
Claims1
A method for increasing fetal hemoglobin levels in a cell, the method comprising the steps of contacting a hematopoietic progenitor cell with an effective amount of a composition comprising an inhibitor of BCL11A, whereby fetal hemoglobin expression is increased in said cell, or its progeny, relative to said cell prior to said contacting.
Dependencies are inferred from the claim text.