Journal
AMERICAN JOURNAL OF HEMATOLOGY
Volume 90, Issue 4, Pages 310-313Publisher
WILEY
DOI: 10.1002/ajh.23934
Keywords
-
Categories
Funding
- Alnylam Pharmaceuticals
Ask authors/readers for more resources
beta-thalassemias result from diminished -globin synthesis and are associated with ineffective erythropoiesis and secondary iron overload caused by inappropriately low levels of the iron regulatory hormone hepcidin. The serine protease TMPRSS6 attenuates hepcidin production in response to iron stores. Hepcidin induction reduces iron overload and mitigates anemia in murine models of -thalassemia intermedia. To further interrogate the efficacy of an RNAi-therapeutic downregulating Tmprss6, -thalassemic Hbb(th3/+) animals on an iron replete, an iron deficient, or an iron replete diet also containing the iron chelator deferiprone were treated with Tmprss6 siRNA. We demonstrate that the total body iron burden is markedly improved in Hbb(th3/+) animals treated with siRNA and chelated with oral deferiprone, representing a significant improvement compared to either compound alone. These data indicate that siRNA suppression of Tmprss6, in conjunction with oral iron chelation therapy, may prove superior for treatment of anemia and secondary iron loading seen in -thalassemia intermedia. Am. J. Hematol. 90:310-313, 2015. (c) 2015 The Authors. American Journal of Hematology Published by Wiley Periodicals, Inc.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available