期刊
HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA
卷 28, 期 2, 页码 387-+出版社
W B SAUNDERS CO-ELSEVIER INC
DOI: 10.1016/j.hoc.2013.11.004
关键词
Iron metabolism; Ineffective erythropoiesis; Hereditary hemochromatosis; beta-Thalassemia; Hepcidin/minihepcidins; Lipid nanoparticle siRNA/antisense oligonucleotide
资金
- Alnylam Pharmaceuticals
- NIH [R01 DK087992, R01 DK100806]
In this article, the authors discuss new approaches to treating iron overload diseases using hepcidin mimetics or by modulating endogenous hepcidin expression. In particular, the authors discuss lipid nanoparticle encapsulated siRNA and antisense oligonucleotide mediated inhibition of TMPRSS6, an upstream regulator of hepcidin, and treatment with transferrin or hepcidin mimetics, including the recently described minihepcidins. In each case, in animal models of beta-thalassemia, not only do the interventions affect iron absorption but they also act as disease-modifying agents that ameliorate the ineffective erythropoiesis.
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