Oral ferroportin inhibitor ameliorates ineffective erythropoiesis in a model of β-thalassemia.

ABSTRACT: β-Thalassemia is a genetic anemia caused by partial or complete loss of β-globin synthesis, leading to ineffective erythropoiesis and RBCs with a short life span. Currently, there is no efficacious oral medication modifying anemia for patients with β-thalassemia. The inappropriately low levels of the iron regulatory hormone hepcidin enable excessive iron absorption by ferroportin, the unique cellular iron exporter in mammals, leading to organ iron overload and associated morbidities. Correction of unbalanced iron absorption and recycling by induction of hepcidin synthesis or treatment with hepcidin mimetics ameliorates β-thalassemia. However, hepcidin modulation or replacement strategies currently in clinical development all require parenteral drug administration. We identified oral ferrop

SUBMITTER: Manolova V 

PROVIDER: S-EPMC6934209 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature