Investigation of curative effect and the neuroplasticity with the start time in the gene therapy of the cerebellar ataxic mouse

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Lentiviral vectors expressing RORα were injected into the cerebellar cortex of P6, P13 or P20 staggerer mice, and the effects were studied 3 weeks after the injection. Staggerer mice lentivirally treated at P6 showed marked dendritic differentiation of PCs and removal CF synapses from PCs, whereas those lentivirally treated at P13 displayed only a partial rescue of dendritic differentiation and failure of removal of CF synapses from PCs. No rescue was observed in staggerer mice lentivirally treated at P20. Together with evidence that protein expression in the brain starts about 5 days after lentiviral vector injection, these results suggest that staggerer mutant cerebellum maintains potential to respond to RORα at 2nd postnatal week, but loses almost completely at 4th postnatal week.