title: Modulation of hepcidin to treat iron deregulation: potential clinical applications.
abstract: The secreted peptide hormone hepcidin regulates systemic and local iron homeostasis through degradation of the iron exporter ferroportin. Dysregulation of hepcidin leads to altered iron homeostasis and development of pathological disorders including hemochromatosis, and iron loading and iron restrictive anemias. Therapeutic modulation of hepcidin is a promising method to ameliorate these conditions. Several approaches have been taken to enhance or reduce the effects of hepcidin in vitro and in vivo. Based on these approaches, hepcidin modulating drugs have been developed and are undergoing clinical evaluation. In this article we review the rationale for development of these drugs, the data concerning their safety and efficacy, their therapeutic uses, and potential future prospects.
authors.affiliation: a Department of Molecular Biology and Biophysics , University of Connecticut Health , Farmington , CT , USA.
authors.forename: Nicole L
authors.initials: NL
authors.lastname: Blanchette
authors.affiliation: a Department of Molecular Biology and Biophysics , University of Connecticut Health , Farmington , CT , USA.|b School of Dental Medicine , University of Connecticut Health , Farmington , CT , USA.
authors.forename: David H
authors.initials: DH
authors.lastname: Manz
authors.affiliation: c Department of Medicine , University of Connecticut Health , Farmington , CT , USA.
authors.forename: Frank M
authors.initials: FM
authors.lastname: Torti
authors.affiliation: a Department of Molecular Biology and Biophysics , University of Connecticut Health , Farmington , CT , USA.
authors.forename: Suzy V
authors.initials: SV
authors.lastname: Torti
mesh_terms: D000818:Animals; D001692:Biological Transport; D052004:Bone Morphogenetic Protein Receptors / Q000037:antagonists & inhibitors / Q000378:metabolism; D019485:Bone Morphogenetic Proteins / Q000378:metabolism; D027682:Cation Transport Proteins / Q000378:metabolism; D005786:Gene Expression Regulation; D064451:Hepcidins / Q000819:agonists / Q000037:antagonists & inhibitors / Q000172:deficiency / Q000378:metabolism*; D006706:Homeostasis; D006801:Humans; D015850:Interleukin-6 / Q000378:metabolism; D007501:Iron / Q000378:metabolism*; D019189:Iron Metabolism Disorders / Q000235:genetics / Q000378:metabolism* / Q000628:therapy*; D036361:Peptide Hormones / Q000494:pharmacology / Q000627:therapeutic use; D015398:Signal Transduction / Q000187:drug effects; D000098742:Ferroportin
chemical_list: D019485:Bone Morphogenetic Proteins; D027682:Cation Transport Proteins; C000600298:Erfe protein, human; D064451:Hepcidins; D015850:Interleukin-6; D036361:Peptide Hormones; D000098742:Ferroportin; D007501:Iron; D052004:Bone Morphogenetic Protein Receptors
keywords: Hepcidin; anemia of chronic disease; clinical trials; ferroportin; iron deficiency; iron overload