An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe(-/-) mice and ameliorates anemia and iron overload in murine β-thalassemia intermedia.

@article{Schmidt2013AnRT,
  title={An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe(-/-) mice and ameliorates anemia and iron overload in murine β-thalassemia intermedia.},
  author={Paul J Schmidt and Iva Toudjarska and Anoop K Sendamarai and Tim Racie and Stuart Milstein and Brian R. Bettencourt and J Hettinger and David A. Bumcrot and Mark D Fleming},
  journal={Blood},
  year={2013},
  volume={121 7},
  pages={1200-8}
}
Mutations in HFE lead to hereditary hemochromatosis (HH) because of inappropriately high iron uptake from the diet resulting from decreased hepatic expression of the iron-regulatory hormone hepcidin. -thalassemia is a congenital anemia caused by partial or complete loss of -globin synthesis causing ineffective erythropoiesis, anemia, decreased hepcidin production, and secondary iron overload. Tmprss6 is postulated to regulate hepcidin production by cleaving Hemojuvelin (Hjv), a key modulator of… CONTINUE READING
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