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FARAFARA Cure FA

Preventing the ubiquitin/proteasome-dependent degradation of frataxin, the protein defective in Friedreich's Ataxia

Friedreich’s Ataxia (FRDA) is a devastating orphan disease, with no specific treatment. The disease is caused by reduced expression of the protein frataxin, which results in mitochondrial defects and oxidative damage. Levels of residual frataxin critically affect onset and progression of the disease. Understanding the molecular mechanisms that regulate frataxin stability and degradation, may therefore be exploited for the design of effective therapeutics.

Preventing the ubiquitin/proteasome-dependent degradation of frataxin, the protein defective in Friedreich's Ataxia


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Jen Farmer

Jen Farmer

Executive Director

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