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Iron-related molecular targets

Molecular classification
Hormone, Transporter, Receptor, Enzyme, Transcription factor, Other
01

Overview

"Iron-related molecular targets" refers to a group of proteins and signaling molecules that collectively regulate systemic and cellular iron homeostasis, a process vital for oxygen transport, DNA synthesis, and energy metabolism (Muckenthaler et al., 2017). The central regulatory axis involves the liver-derived hormone hepcidin and its receptor, the iron exporter ferroportin; hepcidin binding triggers the internalization and degradation of ferroportin, thereby sequestering iron within cells and reducing plasma iron levels (Ganz, 2003). Other significant targets include the transferrin receptor 1 (TfR1), which facilitates cellular iron uptake, and regulatory enzymes such as matriptase-2 (TMPRSS6) and erythroferrone (ERFE) that modulate hepcidin expression in response to iron stores and erythropoietic demand (Finberg, 2008; Kautz et al., 2014). Pharmacological modulation of these targets is a major area of drug development, featuring hepcidin mimetics like rusfertide for polycythemia vera, ferroportin inhibitors like vamifeport for iron-loading anemias, and iron chelators like deferasirox for transfusion-related iron overload (Ginzburg et al., 2023; Richard et al., 2020). Dysregulation of these molecular targets is implicated in a wide range of disorders, including hereditary hemochromatosis, anemia of chronic disease, and various cancers where iron metabolism is hijacked to support rapid proliferation (Camaschella, 2015).

Other names
Iron metabolism regulatorsIron homeostasis proteinsIron-related proteinsIron regulatory networkIron-responsive element-binding proteins
02

Mechanism of action

Modulation of systemic and cellular iron levels through hepcidin agonism or antagonism, ferroportin inhibition, iron chelation, or direct iron supplementation.

03

Biological functions

Iron homeostasisErythropoiesisCellular respirationImmune responseDNA synthesisOther
04

Disease associations

AnemiaHemochromatosisPolycythemia veraChronic kidney diseaseCancerInflammationOther
05

Safety considerations

Iron overload-induced organ damageInfection susceptibility (siderophilic bacteria)Gastrointestinal toxicityHypophosphatemiaHypersensitivity and anaphylaxisOxidative stress
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Interacting drugs

Rusfertide

8 more in the full profile.

07

Biomarkers

Serum ferritinTransferrin saturation (TSAT)Serum hepcidinSoluble transferrin receptor (sTfR)HemoglobinReticulocyte hemoglobin content (CHr)

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