An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe−/− mice and ameliorates anemia and iron overload in murine β-thalassemia intermedia-Reference-Cited by-同舟云学术

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

Published:2013-02-14 Issue:7 Volume:121 Page:1200-1208
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Schmidt Paul J.¹,Toudjarska Iva²,Sendamarai Anoop K.¹,Racie Tim²,Milstein Stuart²,Bettencourt Brian R.²,Hettinger Julia²,Bumcrot David²,Fleming Mark D.¹

Affiliation:

1. Department of Pathology, Children's Hospital Boston and Harvard Medical School, Boston, MA; and

2. Alnylam Pharmaceuticals Inc, Cambridge, MA

Abstract

Key Points Tmprss6 siRNA induces hepcidin and diminishes iron in hemochromatosis or thalassemia mice, improving the anemia seen in the latter model. Manipulation of TMPRSS6 with RNAi therapeutics may be an approach to treating iron overload diseases associated with low hepcidin levels.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/121/7/1200/1368346/zh800713001200.pdf

Reference52 articles.

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3. Blunted hepcidin response to oral iron challenge in HFE-related hemochromatosis.;Piperno;Blood,2007

4. Hfe acts in hepatocytes to prevent hemochromatosis.;Vujić Spasić;Cell Metab,2008

5. Regulatory defects in liver and intestine implicate abnormal hepcidin and Cybrd1 expression in mouse hemochromatosis.;Muckenthaler;Nat Genet,2003

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