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FARAFARA Cure FA

Dimethyl fumarate dosing in humans increases frataxin expression: A potential therapy for Friedreich's Ataxia

High throughput screening of clinically used drugs identified Dimethyl fumarate (DMF) as protective in FA patient cells. This group demonstrates that DMF significantly increases frataxin gene (FXN) expression in FA cell models, FA mouse models and in DMF treated humans. DMF also rescues mitochondrial biogenesis deficiency in FA-patient derived cell models. In FA patient cells, they demonstrate that DMF significantly increases initiation of new FXN transcripts and reduction in DNA structures thought to slow FXN production, significantly increasing FXN expression. Lastly, DMF dosed Multiple Sclerosis (MS) patients showed significant increase in FXN expression by ~85%. As deficiency in FXN is the primary cause of FA, and DMF is demonstrated to increase FXN expression in humans, with further work DMF could be a possible therapy for FA.

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About the Author

Jane Larkindale

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