Several proof-of concept studies have shown promising results in C9orf72 expansions, fused in sarcoma-linked amyotrophic lateral sclerosis, Huntington’s disease and spinocerebellar ataxia type 1, 3, and 7 mouse models and non-human primates as well as in phase I clinical trials90–96. Here, C9orf72 is linked to spinocerebellar ataxia type 1.