MODIFIERS OF GLOBIN GENE EXPRESSION FOR THERAPY OF BETA-THALASSEMIA

The aim of the proposal is to analyse gene expression of erythroid progenitor cells from beta-thalassemia patients, for identification of inducers of fetal hemoglobin of possible interest for therapy of beta-thalassemia. We first will characterize the genotype and the production of fetal hemoglobin in beta-thalassemic patients. After this analysis, we will test the activity of inducers of fetal hemoglobin in erythroid precursor cells isolated from the patients. This will help in identifying the best inducer and in determining whether the studied cells are hydroxyurea-resistant and, in this case, inducible to fetal hemoglobin production with alternative molecules. These informations might be of great interest to propose a pharmacological therapy of beta-thalassemia with the objective to render the patients not dependent from transfusions. The final part of the project is dedicated to exploitation, for identification of Biotech companies interested in our protocols and patents.