Progetto FRRB ID 4976651 -Identification of new strategies for in vivo targeting of erythroid Tfr2 in ß-thalassemia (TARGET-TFR2)
Dati del progetto
Principal Investigator |
Dr.ssa Antonella Nai |
Ente ospitante |
IRCSS Ospedale San Raffaele |
Area Tematica |
Malattie Rare |
Data di inizio Progetto |
02/12/2024 |
Data di fine Progetto |
01/12/2027 |
Importo assegnato |
500.000,00 € |
Tipo Progetto |
Individuale |
PROJECT AIM
Beta-thalassemia is a genetic disease due to mutations in the β-globin gene characterized by anemia, ineffective erythropoiesis, splenomegaly and secondary iron-overload. The management of the disease is still suboptimal and also the most recent approaches are not fully effective in all patients. Transferrin receptor 2 (TFR2) is a novel mediator of the iron-erythropoiesis cross-talk. In hepatocytes, it controls iron homeostasis, while in erythroid cells acts as a brake of erythropoiesis. This project aims at identifying a tool for the downregulation of TFR2 levels/function in erythroid cells in vivo.