The group looked at the neuro-protective effects of the cytokines granulocyte-colony stimulating factor and stem cell factor in a humanized murine model of Friedreich's ataxia. Mice received monthly subcutaneous infusions of cytokines while also being assessed at monthly time points using an extensive range of behavioral motor performance tests. After 6 months of treatment, neurophysiological evaluation of both sensory and motor nerve conduction was performed. Subsequently, mice were sacrificed for mRNA, protein and histological analysis of the dorsal root ganglion, spinal cord and cerebellum. Cytokine administration resulted in significant reversal of biochemical, neuropathological, neurophysiological and behavioral deficits associated with Friedreich's ataxia.
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