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FARAFARA Cure FA

Carbamylated erythropoietin increases frataxin independent from the erythropoietin receptor

Friedreich's ataxia (FRDA) is a neurodegenerative disorder caused by decreased expression of the mitochondrial protein frataxin. Recently we showed in a clinical pilot study in Friedreich's ataxia patients that recombinant human erythropoietin (rhuEPO) significantly increases frataxin-expression. In this in vitro study, we investigated the role of the erythropoietin receptor (EPO-R) in the frataxin increasing effect of rhuEPO and if nonerythropoietic carbamylated erythropoietin (CEPO), which cannot bind to the classical EPO-R increases frataxin expression.

Carbamylated erythropoietin increases frataxin independent from the erythropoietin receptor

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Jen Farmer

Jen Farmer

Executive Director

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