Friedreich's ataxia (FRDA) is the most common hereditary ataxia, affecting approximately 3 in 100,000 individuals in Caucasian populations. It is caused by intronic GAA repeat expansions that hinder the expression of the FXN gene, resulting in defective levels of the mitochondrial protein frataxin. Sensory neurons in dorsal root ganglia (DRG) are particularly damaged by frataxin deficiency. There is no specific therapy for FRDA.
Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model
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