EHA 2018 | Revolutionary approaches for thalassemia

Maria D. Cappellini, Maria D. Cappellini and Maria Domenica Cappellini

Thalassemia is caused by defects in either α or β 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 α and β 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.

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