EHA 2019: Thalassemia: Double targeting of abnormal erythropoiesis strikingly improves anemia

Antonella Nai | EHA 2019 | Juni 17, 2019

Speaker: Antonella Nai

Beta-thalassemiasare inherited disorders that cause severe impairment of red cell production, anemia, iron overload and organ damage.In this MEDtalkDr Antonella Naiexplain, the clinical benefitin combination of the bone marrow-specific deletion of the erythropoiesis regulator TFR2, leading to an improved erythropoietin response, and with oligonucleotides that, degrading the product of the iron regulatory gene TMPRSS6, decrease the excess iron.