Thalassemia is caused by defects in either ? or ? globin chains, which are required for formation of functional hemoglobin molecules. Maria Domenica Cappellini, MD, of the University of Milan-Foundation IRCCS Policlinico Hospital, Milan, Italy, spoke to us at the 23rd Congress of European Hematology Association (EHA) 2018, held in Stockholm, Sweden, about new treatments that seek to re-establish the balance between ? and ? chains to reduce ineffective erythropoiesis. Techniques for achieving these aims include: genome editing to increase production of fetal hemoglobin; correcting ineffective erythropoiesis using ACE inhibitors; and gene therapy.
Revolutionary approaches for thalassemia
