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Successful treatment for mice with beta-thalassemia

Date:
November 22, 2010
Source:
Journal of Clinical Investigation
Summary:
Beta-thalassemia is an inherited blood disorder that results in chronic anemia. A major complication of the condition is iron overload, which has been linked to low levels of the protein hepcidin. New research indicates that increasing the concentration of hepcidin in beta-thalassemic mice limits iron overload and markedly reduces their anemia, pointing to a potential new therapeutic approach to limiting iron overload and mitigating anemia in patients with beta-thalassemia.
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Beta-thalassemia is an inherited blood disorder that results in chronic anemia. A major complication of the condition is iron overload, which damages organs such as the liver and heart. The iron overload has been linked to low levels of the protein hepcidin, a negative regulator of intestinal iron absorption and iron recycling.

A team of researchers, led by Stefano Rivella, at Weill Cornell Medical College, New York, has now shown that increasing the concentration of hepcidin in beta-thalassemic mice limits iron overload and markedly reduces their anemia. They therefore suggest that therapeutic approaches that increase hepcidin levels in patients with beta-thalassemia could be therapeutic, limiting iron overload and mitigating anemia.

In an accompanying commentary, Mark Fleming and Thomas Bartnikas, at Children's Hospital Boston, discuss these data and suggest that modulating hepcidin levels could be a new approach to treating a multitude of diseases associated with iron overload or deficiency.


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The above post is reprinted from materials provided by Journal of Clinical Investigation. Note: Materials may be edited for content and length.


Journal Reference:

  1. Sara Gardenghi, Pedro Ramos, Maria Franca Marongiu, Luca Melchiori, Laura Breda, Ella Guy, Kristen Muirhead, Niva Rao, Cindy N. Roy, Nancy C. Andrews, Elizabeta Nemeth, Antonia Follenzi, Xiuli An, Narla Mohandas, Yelena Ginzburg, Eliezer A. Rachmilewitz, Patricia J. Giardina, Robert W. Grady, Stefano Rivella. Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice. Journal of Clinical Investigation, 2010; DOI: 10.1172/JCI41717

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Journal of Clinical Investigation. "Successful treatment for mice with beta-thalassemia." ScienceDaily. ScienceDaily, 22 November 2010. <www.sciencedaily.com/releases/2010/11/101122121633.htm>.
Journal of Clinical Investigation. (2010, November 22). Successful treatment for mice with beta-thalassemia. ScienceDaily. Retrieved August 3, 2015 from www.sciencedaily.com/releases/2010/11/101122121633.htm
Journal of Clinical Investigation. "Successful treatment for mice with beta-thalassemia." ScienceDaily. www.sciencedaily.com/releases/2010/11/101122121633.htm (accessed August 3, 2015).

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