Combinatorial compositions and methods of treating hemoglobinopathies
AssigneeChildren's Medical Center Corporation
InventorsStuart H. Orkin, Jian Xu, Peng Cong
Life of this patent
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Abstract
Provided herein are compositions and methods for increasing fetal hemoglobin expression (HbF) for the treatment and/or amelioration of the symptoms of a hemoglobinpathy in a mammal. The compositions comprise a combination of one or more inhibitors of BCL11A expression or activity with epigenetic modifiers, such as inhibitors of DNA methylation and/or one or more inhibitors of histone deacetylases.
Claims20
A pharmaceutical composition for increasing fetal hemoglobin levels in a mammal in need thereof, the composition comprising at least one inhibitor of BCL11A and at least one epigenetic modifier in a pharmaceutically acceptable carrier.
-25. (canceled)
Dependencies are inferred from the claim text.