Thalassemia: Double targeting of abnormal erythropoiesis strikingly improves anemia

BestPractice | Jun 2019 | |

Beta-thalassemias are inherited disorders that cause severe impairment of red cell production, anemia, iron overload and organ damage. In this MEDtalk Dr Antonella Nai explain, the clinical benefit in 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.