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An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe(-/-) mice and ameliorates anemia and iron overload in murine β-thalassemia intermedia.

Schmidt PJ, Toudjarska I, Sendamarai AK, Racie T, Milstein S, Bettencourt BR, Hettinger J, Bumcrot D, Fleming MD.

Blood. 2013 Feb 14;121(7):1200-8. doi: 10.1182/blood-2012-09-453977. Epub 2012 Dec 6.


Minihepcidins prevent iron overload in a hepcidin-deficient mouse model of severe hemochromatosis.

Ramos E, Ruchala P, Goodnough JB, Kautz L, Preza GC, Nemeth E, Ganz T.

Blood. 2012 Nov 1;120(18):3829-36. doi: 10.1182/blood-2012-07-440743. Epub 2012 Sep 18.


The murine growth differentiation factor 15 is not essential for systemic iron homeostasis in phlebotomized mice.

Casanovas G, Vujić Spasic M, Casu C, Rivella S, Strelau J, Unsicker K, Muckenthaler MU.

Haematologica. 2013 Mar;98(3):444-7. doi: 10.3324/haematol.2012.069807. Epub 2012 Sep 14.


Neogenin interacts with matriptase-2 to facilitate hemojuvelin cleavage.

Enns CA, Ahmed R, Zhang AS.

J Biol Chem. 2012 Oct 12;287(42):35104-17. doi: 10.1074/jbc.M112.363937. Epub 2012 Aug 14.


The definition and epidemiology of non-transfusion-dependent thalassemia.

Weatherall DJ.

Blood Rev. 2012 Apr;26 Suppl 1:S3-6. doi: 10.1016/S0268-960X(12)70003-6. Review.


Contemporary approaches to treatment of beta-thalassemia intermedia.

Taher AT, Musallam KM, Karimi M, Cappellini MD.

Blood Rev. 2012 Apr;26 Suppl 1:S24-7. doi: 10.1016/S0268-960X(12)70008-5. Review.


The role of ineffective erythropoiesis in non-transfusion-dependent thalassemia.

Rivella S.

Blood Rev. 2012 Apr;26 Suppl 1:S12-5. doi: 10.1016/S0268-960X(12)70005-X. Review.


Recent advances and treatment challenges in patients with non-transfusion-dependent thalassemia.

Taher AT, Cappellini MD, Musallam KM.

Blood Rev. 2012 Apr;26 Suppl 1:S1-2. doi: 10.1016/S0268-960X(12)00028-8. No abstract available.


Deletion of TMPRSS6 attenuates the phenotype in a mouse model of β-thalassemia.

Nai A, Pagani A, Mandelli G, Lidonnici MR, Silvestri L, Ferrari G, Camaschella C.

Blood. 2012 May 24;119(21):5021-9. doi: 10.1182/blood-2012-01-401885. Epub 2012 Apr 6.


Decreased hepcidin expression in murine β-thalassemia is associated with suppression of Bmp/Smad signaling.

Parrow NL, Gardenghi S, Ramos P, Casu C, Grady RW, Anderson ER, Shah YM, Li H, Ginzburg YZ, Fleming RE, Rivella S.

Blood. 2012 Mar 29;119(13):3187-9. doi: 10.1182/blood-2012-01-405563. No abstract available.


Stimulated erythropoiesis with secondary iron loading leads to a decrease in hepcidin despite an increase in bone morphogenetic protein 6 expression.

Frazer DM, Wilkins SJ, Darshan D, Badrick AC, McLaren GD, Anderson GJ.

Br J Haematol. 2012 Jun;157(5):615-26. doi: 10.1111/j.1365-2141.2012.09104.x. Epub 2012 Mar 26.


Minihepcidins are rationally designed small peptides that mimic hepcidin activity in mice and may be useful for the treatment of iron overload.

Preza GC, Ruchala P, Pinon R, Ramos E, Qiao B, Peralta MA, Sharma S, Waring A, Ganz T, Nemeth E.

J Clin Invest. 2011 Dec;121(12):4880-8. doi: 10.1172/JCI57693.


Natural history and management of HFE-hemochromatosis.

Gan EK, Powell LW, Olynyk JK.

Semin Liver Dis. 2011 Aug;31(3):293-301. doi: 10.1055/s-0031-1286060. Epub 2011 Sep 7. Review.


Perturbation of hepcidin expression by BMP type I receptor deletion induces iron overload in mice.

Steinbicker AU, Bartnikas TB, Lohmeyer LK, Leyton P, Mayeur C, Kao SM, Pappas AE, Peterson RT, Bloch DB, Yu PB, Fleming MD, Bloch KD.

Blood. 2011 Oct 13;118(15):4224-30. doi: 10.1182/blood-2011-03-339952. Epub 2011 Aug 12.


β-thalassemia: a model for elucidating the dynamic regulation of ineffective erythropoiesis and iron metabolism.

Ginzburg Y, Rivella S.

Blood. 2011 Oct 20;118(16):4321-30. doi: 10.1182/blood-2011-03-283614. Epub 2011 Jul 18. Review.


Prospects for a hepcidin mimic to treat β-thalassemia and hemochromatosis.

Parrow NL, Gardenghi S, Rivella S.

Expert Rev Hematol. 2011 Jun;4(3):233-5. doi: 10.1586/ehm.11.22. No abstract available.


Preparation of isolated rat liver hepatocytes.

Quistorff B, Dich J, Grunnet N.

Methods Mol Biol. 1990;5:151-60. doi: 10.1385/0-89603-150-0:151.


Tmprss6 is a genetic modifier of the Hfe-hemochromatosis phenotype in mice.

Finberg KE, Whittlesey RL, Andrews NC.

Blood. 2011 Apr 28;117(17):4590-9. doi: 10.1182/blood-2010-10-315507. Epub 2011 Feb 25.


Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice.

Gardenghi S, Ramos P, Marongiu MF, Melchiori L, Breda L, Guy E, Muirhead K, Rao N, Roy CN, Andrews NC, Nemeth E, Follenzi A, An X, Mohandas N, Ginzburg Y, Rachmilewitz EA, Giardina PJ, Grady RW, Rivella S.

J Clin Invest. 2010 Dec;120(12):4466-77. doi: 10.1172/JCI41717. Epub 2010 Nov 22.


Anemia, ineffective erythropoiesis, and hepcidin: interacting factors in abnormal iron metabolism leading to iron overload in β-thalassemia.

Gardenghi S, Grady RW, Rivella S.

Hematol Oncol Clin North Am. 2010 Dec;24(6):1089-107. doi: 10.1016/j.hoc.2010.08.003. Epub 2010 Oct 15. Review.

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