TMPRSS6 as a Therapeutic Target for Disorders of Erythropoiesis and Iron Homeostasis
Tomas Ganz, Elizabeta Nemeth, Stefano Rivella, Paul Goldberg, Andrew R. Dibble, Michael L. McCaleb, Shuling Guo, Brett P. Monia, Terrance D. Barrett
Abstract
TMPRSS6 is a serine protease highly expressed in the liver. Its role in iron regulation was first reported in 2008 when mutations in TMPRSS6 were shown to be the cause of iron-refractory iron deficiency anemia (IRIDA) in humans and in mouse models. TMPRSS6 functions as a negative regulator of the expression of the systemic iron-regulatory hormone hepcidin. Over the last decade and a half, growing understanding of TMPRSS6 biology and mechanism of action has enabled development of new therapeutic approaches for patients with diseases of erythropoiesis and iron homeostasis.ClinicalTrials.gov identifier NCT03165864.