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Spinocerebellar ataxia type 3 (SCA3) is caused by the expansion of a CAG repeat tract that affects the  MJD1  gene which encodes the ataxin-3 protein. In order to analyze whether symptoms caused by ataxin-3 with an expanded repeat are reversible  in vivo , we generated a conditional mouse model of SCA3 using the Tet-Off system. We used a full-length human  ataxin-3  cDNA with 77 repeats in order to generate the responder mouse line. After crossbreeding with a  PrP  promoter mouse line, double transgenic mice developed a progressive neurological phenotype characterized by neuronal dysfunction in the cerebellum, reduced anxiety, hyperactivity, impaired Rotarod performance and lower body weight gain. When ataxin-3 expression was turned off in symptomatic mice in an early disease state, the transgenic mice were indistinguishable from negative controls after 5 months of treatment. These results show that reducing the production of pathogenic ataxin-3 indeed may be a promising approach to treat SCA3, provided that such treatment is applied before irreversible damage has taken place and that it is continued for a sufficiently long time.

Reversibility of symptoms in a conditional mouse model of spinocerebellar ataxia type 3