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2014 ; 28
(2
): 387-401
Nephropedia Template TP
gab.com Text
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Modulation of hepcidin as therapy for primary and secondary iron overload
disorders: preclinical models and approaches
#MMPMID24589273
Schmidt PJ
; Fleming MD
Hematol Oncol Clin North Am
2014[Apr]; 28
(2
): 387-401
PMID24589273
show ga
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
?-thalassemia, not only do the interventions affect iron absorption but they also
act as disease-modifying agents that ameliorate the ineffective erythropoiesis.