Indeed, the observation that treatment with the NEK6 inhibitor, inhibitor 8, rescued axonal transport defects in iPSC-derived cortical neurons both following DPR treatment and from C9orf72 patients, supports the notion that downregulating NEK6 levels/activity has a protective effect, in contrast to the harmful effects of decreased NEK1 levels shown in ALS.37 Therefore, NEK6 may play a crucial regulatory role in C9orf72-related toxicity. This evidence concerns the gene C9orf72 and amyotrophic lateral sclerosis.