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Current experiments rely on FXN-knockdown cells or YG8s mouse models, which do not replicate heterogeneity in GAA repeat expansion length observed in patients (a factor strongly correlated with disease severity) and do not evaluate organ-targeting efficiency or long-term safety of ferroptosis inhibitors such as deferiprone—particularly whether simultaneous delivery to cerebellum and heart is achievable and whether sustained treatment disrupts erythroid iron homeostasis and induces anemia

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[DOI] [PubMed] [Google Scholar] 49.Dohner K, Wolfstein A, Prank U, Echeverri C, Dujardin D, Vallee R, et al