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Deferiprone for the treatment of Friedreich's ataxia
Author(s) -
Pandolfo Massimo,
Hausmann Laura
Publication year - 2013
Publication title -
journal of neurochemistry
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.75
H-Index - 229
eISSN - 1471-4159
pISSN - 0022-3042
DOI - 10.1111/jnc.12300
Subject(s) - frataxin , deferiprone , ataxia , biogenesis , iron–sulfur cluster , context (archaeology) , deferoxamine , mitochondrion , chemistry , biochemistry , iron homeostasis , transferrin , microbiology and biotechnology , iron binding proteins , biology , neuroscience , metabolism , enzyme , paleontology , gene
Abstract Friedreich's ataxia ( FRDA ) is a neurological disease related to a deficiency of the protein frataxin involved in iron–sulfur (Fe–S) cluster biogenesis. This leads to an increased cellular iron uptake accumulating in mitochondria, and a subsequently disturbed iron homeostasis. The detailed mechanism of iron regulation of frataxin expression is yet unknown. Deferiprone, an iron chelator that may cross the blood–brain barrier, was shown to shuttle iron between subcellular compartments. It could also transfer iron from iron‐overloaded cells to extracellular apotransferrin and pre‐erythroid cells for heme synthesis. Here, clinical studies on Deferiprone are reviewed in the context of alternative agents such as desferoxamine, with specific regard to its mechanistic and clinical implications.