PROGETTO TARGET-TFR2

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.