RAS RESPONSIVE ELEMENT BINDING PROTEIN 1 (RREB1) AS A THERAPEUTIC TARGET FOR THALASSEMIAS AND SICKLE CELL ANEMIA

摘要

A method of screening compounds capable of activating ζ and/or &egr; globin gene promoter activity in an erythroid cell is disclosed. The methods comprises contacting in a medium a compound to be screened with RREB1; wherein the medium comprises a polynucleotide comprising the nucleotide sequence of 5′-M-C-M-C-A-M-M-H-M-M-M-3′, wherein M is the nucleotide adenine or cytosine, and H is the nucleotide adenine, cytosine or thymine; the RREB1 bindable to the polynucleotide; determining binding of the compound to the RREB1; and determining change in binding of the RREB1 to the polynucleotide; wherein detection of binding of the compound to the RREB1 and change in binding of the RREB1 to the polynucleotide is indicative that the compound is capable of activating ζ and/or &egr; globin gene promoter activity. Also disclosed is a method of activating ζ and/or &egr; globin gene promoter activity in an erythroid cell.