Friedreich ataxia is a hereditary disease caused by mutation of the frataxin (FXN) gene and resultant low levels of the FXN protein. In a mouse model of the disease, Tomassini et al. found that subcutaneous injections of interferon-γ for 14 weeks restored FXN levels in dorsal root ganglion neurons, which are particularly vulnerable in this disease, and improved motor coordination. The findings open therapeutic avenues for Friedreich ataxia, a disease for which no specific treatments are currently available.
ORIGINAL RESEARCH PAPER
Tomassini, B. et al. Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model. Hum. Mol. Genet. doi:10.1093/hmg/dds110
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Interferon-γ shows promise in a mouse model of Friedreich ataxia. Nat Rev Neurol 8, 242 (2012). https://doi.org/10.1038/nrneurol.2012.74
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DOI: https://doi.org/10.1038/nrneurol.2012.74