A mouse model for DRPLA, based on transgenic expression throughout the brain with the PrP promoter of a mutant human ATN-1 encoding for a 65Q expansion closely recapitulates the late-onset DRPLA with predominantly cerebellar degeneration, ataxic gait, anxiety and premature death17. This evidence concerns the gene ATN1 and dentatorubral-pallidoluysian atrophy.