Although a tendency was observed for an age-related aggravation of the phenotype between 8 and 12 weeks after LV-MUT-ATXN7 injection, the difference in performance between 8 and 12 weeks did not reach statistical significance (Fig. 5c and d ).
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Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins.
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