Transferrin therapy ameliorates disease in Β-thalassemic mice

Huihui Li, Anne C. Rybicki, Sandra M. Suzuka, Leni Von Bonsdorff, William Breuer, Charles B. Hall, Z. Ioav Cabantchik, Eric E. Bouhassira, Mary E. Fabry, Yelena Z. Ginzburg

Research output: Contribution to journalArticlepeer-review

163 Scopus citations


Individuals with Β-thalassemia develop progressive systemic iron overload, resulting in high morbidity and mortality. These complications are caused by labile plasma iron, which is taken up by parenchymal cells in a dysregulated manner; in contrast, erythropoiesis depends on transferrin-bound iron uptake via the transferrin receptor. We hypothesized that the ineffective erythropoiesis and anemia observed in Β-thalassemia might be ameliorated by increasing the amount of circulating transferrin. We tested the ability of transferrin injections to modulate iron metabolism and erythropoiesis in Hbb th1/th1 mice, an experimental model of Β-thalassemia. Injected transferrin reversed or markedly improved the thalassemia phenotype in these mice. Specifically, transferrin injections normalized labile plasma iron concentrations, increased hepcidin expression, normalized red blood cell survival and increased hemoglobin production; this treatment concomitantly decreased reticulocytosis, erythropoietin abundance and splenomegaly. These results indicate that transferrin is a limiting factor contributing to anemia in these mice and suggest that transferrin therapy might be beneficial in human Β-thalassemia.

Original languageEnglish (US)
Pages (from-to)177-182
Number of pages6
JournalNature Medicine
Issue number2
StatePublished - Feb 2010

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)


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