Novel therapy for disorders of iron overload

DESCRIPTION (provided by applicant): Iron-overload disorders, including hereditary hemochromatosis, are typically insidious, causing progressive and sometimes irreversible end-organ injury before clinical symptoms develop. Iron overload in vital organs increases the risk for liver disease (cirrhosis, cancer), heart attack or heart failure, diabetes mellitus, osteoarthrtis, osteoporosis, metabolic syndrome, and other disorders. Iron overload can be inherited or acquired by receiving numerous blood transfusions or consuming high levels of supplemental iron. ?-Thalassemia and HFE-related hemochromatosis are two of the most frequently inherited disorders worldwide. Both disorders are characterized by low levels of hepcidin (HAMP), the hormone that regulates ironabsorption. Routine treatment in an otherwise-healthy person consists of regularly scheduled phlebotomies or iron-chelating agents like deferoxamine. Most types of iron overload are associated with low levels of hepcidin, a peptide that regulates iron met