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Mitapivat metabolically reprograms human β-thalassemic erythroblasts, increasing their responsiveness to oxidation

Abstract: β-thalassemia (β-thal) is a worldwide hereditary red cell disorder characterized by severe chronic anemia. Recently, the pyruvate kinase (PK) activator mitapivat has been shown to improve anemia and ineffective erythropoiesis in a mouse model of β-thal and in patients with non–transfusion-...

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Autori principali: Angela Siciliano, Angelo D’Alessandro, Alessandro Matte, Giovanni Bisello, Mariarita Bertoldi, Monika Dzieciatkowska, Amy Argabright, Richard Huot Pozzetto, Veronica Riccardi, Andrea Mattarei, Alberto Ongaro, Jacopo Ceolan, Roberta Russo, Leonardo Rivadeneyra, Megan Wind-Rotolo, Achille Iolascon, Carlo Brugnara, Lucia De Franceschi
Natura: Artigo
Lingua:Inglês
Pubblicazione: Elsevier 2025-06-01
Serie:Blood Advances
Accesso online:http://www.sciencedirect.com/science/article/pii/S2473952925001636
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