Finally, we confirmed C9orf72-135’s activity in an additional series of cellular disease models: C9-ALS-iPSC-derived cortical neurons (denoted ALS cortical neurons), C9-ALS patient-derived fibroblasts and primary neurons from C9BAC transgenic mice29 (Supplementary Fig. 2a–e). This evidence concerns the gene C9orf72 and amyotrophic lateral sclerosis.