4.8 Editorial Material

Striking the target in iron overload disorders

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JOURNAL OF CLINICAL INVESTIGATION
卷 123, 期 4, 页码 1424-1427

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AMER SOC CLINICAL INVESTIGATION INC
DOI: 10.1172/JCI68889

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  1. NIDDK NIH HHS [K08 DK084204] Funding Source: Medline

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The liver, a major site of body iron stores, mediates key responses that preserve systemic iron homeostasis. In this issue of the JCI, Guo et al. demonstrate that administration of antisense oligonudeotides that reduce expression of Tmprss6, a hepatic protein that plays an essential role in maintaining iron balance, can attenuate disease severity in mouse models of human iron overload disorders. These data reveal the potential of novel TMPRSS6-targeted therapies for the treatment of clinical conditions such as hereditary hemochromatosis and beta-thalassemia.

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