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A preclinical approach for gene therapy of beta-thalassemia.
Academic Article
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Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia.
Academic Article
Why?
Hepcidin and Hfe in iron overload in beta-thalassemia.
Academic Article
Why?
Low bone mineral density in adolescents with beta-thalassemia.
Academic Article
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Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy.
Academic Article
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Strategy for a multicenter phase I clinical trial to evaluate globin gene transfer in beta-thalassemia.
Academic Article
Why?
beta-Thalassemia
Concept
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A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia.
Academic Article
Why?
Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial.
Academic Article
Why?
Ineffective erythropoiesis in beta-thalassemia is characterized by increased iron absorption mediated by down-regulation of hepcidin and up-regulation of ferroportin.
Academic Article
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Wolfe, Scot
Person
Why?
Exploring the role of hepcidin, an antimicrobial and iron regulatory peptide, in increased iron absorption in beta-thalassemia.
Academic Article
Why?
Combined iron chelation therapy.
Academic Article
Why?
Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial.
Academic Article
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Genomic safe harbors permit high ?-globin transgene expression in thalassemia induced pluripotent stem cells.
Academic Article
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