Hepcidin-regulatory pathway gene mRNA

Hepcidin-regulatory pathway gene mRNA refers to the messenger RNA transcripts of genes that control the expression of hepcidin, the primary hormone regulating systemic iron balance. The pathway includes positive regulators like HAMP (hepcidin), HFE, and TFR2, as well as negative regulators such as TMPRSS6 (matriptase-2). These mRNAs are targeted by RNA-based therapeutics, including antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), to either increase or decrease hepcidin levels. For example, silencing TMPRSS6 mRNA leads to increased hepcidin, which restricts iron absorption and is used to treat iron overload in conditions like beta-thalassemia and polycythemia vera. Conversely, targeting HAMP mRNA directly can lower hepcidin levels to alleviate iron restriction in anemia of chronic disease. These therapies are typically delivered to the liver using GalNAc-conjugation to ensure high specificity and potency.

Other names

HAMP-regulatory mRNAHepcidin pathway mRNATMPRSS6 mRNAHAMP mRNATFR2 mRNAMatriptase-2 mRNA

Antisense inhibition or RNA interference-mediated degradation of target mRNA transcripts to modulate the expression of proteins in the hepcidin-regulatory pathway, thereby controlling systemic iron levels.