ATXN1 and spinocerebellar ataxia type 1: Intracerebroventricular (ICV) injection of a non-allele specific gapmer resulted in a robust decrease of Atxn1 mRNA and ATXN1 protein expression in cerebellum, cortex, pons, and medulla in a SCA1 mouse model—Atxn1154Q/2Q knock-in mouse model [176].