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

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Presentation on theme: "An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe −/− mice and ameliorates anemia and iron overload in murine β-thalassemia intermedia."— Presentation transcript:

1 An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe −/− mice and ameliorates anemia and iron overload in murine β-thalassemia intermedia by Paul J. Schmidt, Iva Toudjarska, Anoop K. Sendamarai, Tim Racie, Stuart Milstein, Brian R. Bettencourt, Julia Hettinger, David Bumcrot, and Mark D. Fleming Blood Volume 121(7):1200-1208 February 14, 2013 ©2013 by American Society of Hematology

2 Analysis of hepcidin expression and serum transferrin saturation in WT mice treated with an RNAi therapeutic directed against Tmprss6. Paul J. Schmidt et al. Blood 2013;121:1200-1208 ©2013 by American Society of Hematology

3 Phenotypic analysis of a HH mouse (Hfe−/−) model after treatment after treatment with LNP- Tmprss6 siRNA. Paul J. Schmidt et al. Blood 2013;121:1200-1208 ©2013 by American Society of Hematology

4 Analysis of erythropoietic parameters in treated Hfe−/− mice. Paul J. Schmidt et al. Blood 2013;121:1200-1208 ©2013 by American Society of Hematology

5 Phenotypic analysis of a thalassemia-intermedia mouse (Hbbth3/+) model after treatment with LNP-Tmprss6 siRNA. Paul J. Schmidt et al. Blood 2013;121:1200-1208 ©2013 by American Society of Hematology

6 Analysis of erythropoietic parameters in treated Hbbth3/+ mice. Paul J. Schmidt et al. Blood 2013;121:1200-1208 ©2013 by American Society of Hematology

7 Analysis of RBC maturation in treated Hbbth3/+ mice. Paul J. Schmidt et al. Blood 2013;121:1200-1208 ©2013 by American Society of Hematology


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