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EHA 2019 | New double targeting approach for beta-thalassemia

Antonella Nai of San Raffaele Scientific Institute, Milan, Italy, discusses the double targeting of abnormal erythropoiesis in a thalassemia mouse model, which was demonstrated to strikingly improve anemia. In this preclinical study on a thalassemic mouse mode, two treatments that have previously been shown to individually improve anemia were combined. The bone marrow-specific deletion of TFR2 was combined with oligonucleotides that degrade the product of TMPRSS6. This press briefing was recorded at the 24th Congress of the European Hematology Association (EHA) 2019, held in Amsterdam, Netherlands.

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