Thalassemia and B and D Globin Gene Expression

The approach of Oncogene Science for the treatment of beta-thalassemia is to identify chemical compmodulate transcription of the adult beta-globin gene and/or induce expression of the delta-globin geselection is considerably facilitated by the use of highly sophisticated automated facility which pescreening of about 120,000 compounds per year against a single therapeutic target. A large body of dhypothesis that the globin genes compete for a limited number of activating transcription factors. Tglobin promoter activity has been shown to correlate with increased transcription of the delta and gtreatment of severe beta-thalassemia involving homozygous structural alteration of the beta-globin gbeta-globin promoter activity is predicted to result in increased gamma and delta-globin synthesis aproduction, and alleviate the otherwise fatal symptoms of beta- thalassemia. The delta and beta promLCR HS-II element, will be fused to the luciferase reporter gene. In addition, the luciferase gene wtranslational initiation codon (ATG) of the two respective genes, within the context of a plasmid habeta locus. In Phase II, these stable cell lines will be employed in high throughput robotic drug scwhich modulate expression of the beta and delta globin genes, in subsequent assays of selectivity anmechanism of drug action.