Modulation of hepcidin as therapy for primary and secondary iron overload disorders: preclinical models and approaches.

Abstract

In this article, the authors discuss new approaches to treating iron overload diseases using hepcidin mimetics or by modulating endogenous hepcidin expression. In particular, the authors discuss lipid nanoparticle encapsulated siRNA and antisense oligonucleotide-mediated inhibition of TMPRSS6, an upstream regulator of hepcidin, and treatment with transferrin or hepcidin mimetics, including the recently described minihepcidins. In each case, in animal models of β-thalassemia, not only do the interventions affect iron absorption but they also act as disease-modifying agents that ameliorate the ineffective erythropoiesis.

• Authors: Paul J Schmidt, Mark D Fleming
• Journal: Hematology/oncology clinics of North America (Hematol Oncol Clin North Am) Vol. 28 Issue 2 Pg. 387-401 (Apr 2014) ISSN: 1558-1977 [Electronic] United States
• PMID: 24589273 (Publication Type: Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't, Review)
• Copyright: Copyright © 2014 Elsevier Inc. All rights reserved.

Chemical References

• Hepcidins
• Transferrin
• Genistein
• Iron

Topics

• Animals
• Disease Models, Animal
• Gene Expression (drug effects)
• Genistein (pharmacology, therapeutic use)
• Hepcidins (genetics, metabolism)
• Humans
• Iron (metabolism)
• Iron Overload (drug therapy, metabolism)
• Transferrin (pharmacology, therapeutic use)
• beta-Thalassemia (drug therapy, genetics, metabolism)