Breda Laura, Gardenghi Sara, Guy Ella, Rachmilewitz Eliezer A, Weizer-Stern Orly, Adamsky Konstantin, Amariglio Ninette, Rechavi Gideon, Giardina Patricia J, Grady Robert W, Rivella Stefano
Weill Medical College of Cornell University, Department of Pediatrics, Division of Hematology-Oncology, Children's Blood Foundation Laboratories, New York, New York 10021, USA.
Ann N Y Acad Sci. 2005;1054:417-22. doi: 10.1196/annals.1345.069.
To develop new treatments for beta-thalassemia, it is essential to identify the genes involved in the relevant pathophysiological processes. Iron metabolism in thalassemia mice being investigated, focusing on the expression of a gene called hepcidin (Hamp), which is expressed in the liver and whose product (Hamp) is secreted into the bloodstream. In mice, iron overload leads to overexpression of Hamp, while Hamp-knockout mice suffer from hemochromatosis. The aim of this study is to investigate Hamp in the mouse model of beta-thalassemia and to address the potential gene transfer of Hamp to prevent abnormal iron absorption.
为了开发β地中海贫血的新疗法，确定参与相关病理生理过程的基因至关重要。正在研究地中海贫血小鼠的铁代谢，重点关注一种名为铁调素（Hamp）的基因的表达，该基因在肝脏中表达，其产物（Hamp）分泌到血液中。在小鼠中，铁过载会导致Hamp过度表达，而Hamp基因敲除小鼠会患血色素沉着症。本研究的目的是在β地中海贫血小鼠模型中研究Hamp，并探讨Hamp的潜在基因转移以防止异常铁吸收。
