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Use of modulators of compounds of the TGF-β superfamily to control iron metabolism by hepcidin

机译:TGF-β超家族化合物的调节剂在铁调素控制铁代谢中的应用

摘要

The present invention provides new systems and strategies for the regulation of iron metabolism in mammals. In particular, methods of using agonists and antagonists of TGF-² superfamily members to modulate the expression or activity of hepcidin, a key regulator of iron metabolism, are described. The inventive methods find applications in the treatment of diseases associated with iron overload, such as juvenile hemochromatosis and adult hemochromatosis, and in the treatment of diseases associated with iron deficiency, such as anemia of chronic disease and EPO resistant anemia in end-stage of renal disease. The present invention also relates to screening tools and methods for the development of novel drugs and therapies for treating iron metabolism disorders.
机译:本发明提供了用于调节哺乳动物中铁代谢的新系统和策略。特别地,描述了使用TGF-β2超家族成员的激动剂和拮抗剂来调节铁代谢的关键调节剂铁调素的表达或活性的方法。本发明的方法可用于治疗与铁超负荷有关的疾病,例如青少年血色素沉着症和成人血色素沉着症;以及与铁缺乏症有关的疾病,例如慢性疾病性贫血和肾末期的EPO耐药性贫血。疾病。本发明还涉及用于开发用于治疗铁代谢异常的新药和疗法的筛选工具和方法。

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