Modulation of Hepcidin as Therapy for Primary and Secondary Iron Overload Disorders: Preclinical Models and Approaches

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