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.
Claims27
A CD34+ hematopoietic progenitor cell that comprises a vector encoding a nucleic acid that inhibits BCL11A expression.
A composition comprising a CD34+ hematopoietic progenitor cell that comprises a viral vector encoding a nucleic acid that inhibits BCL11A expression.
A population of CD34+ hematopoietic progenitor cells, wherein the CD34+ hematopoietic progenitor cells comprise a viral vector encoding a nucleic acid that inhibits BCL11A expression.
A composition comprising a population of CD34+ hematopoietic progenitor cells that comprises a viral vector encoding a nucleic acid that inhibits BCL11A expression.
Dependencies are inferred from the claim text.