Use of modulators of compounds of tgf-beta superfamily to...

A - Human Necessities – 61 – K

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A61K 38/17 (2006.01) A61K 39/395 (2006.01) A61K 47/48 (2006.01) A61P 3/02 (2006.01) A61P 7/06 (2006.01)

Patent

CA 2597925

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-.beta. 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.

La présente invention concerne des systèmes et des stratégies nouveaux pour la régulation du métabolisme du fer chez les mammifères. L'invention concerne notamment des méthodes d'utilisation d'agonistes et d'antagonistes de membres de la superfamille de TGF-ß afin de moduler l'expression ou l'activité de l'hepcidine, régulateur clé du métabolisme du fer. Les méthodes de l'invention trouvent des applications dans le traitement de maladies associées à une surcharge en fer, telle que l'hémochromatose juvénile et l'hémochromatose adulte, ainsi que dans le traitement de maladies associées à une carence en fer, telle que l'anémie d'une maladie chronique et l'anémie résistante à l'EPO à un stade terminal d'une maladie rénale. La présente invention concerne enfin des outils de criblage et des méthodes pour le développement de médicaments et de thérapies nouveaux destinés au traitement de troubles du métabolisme du fer.

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