Importantly, our findings from various cell models, including C9orf72-ALS patient–derived iPSCs, motor neurons, and the repeat expansion mouse model, demonstrate that the reduction of RAN translation products by treatment with the CRISPR/Cas13d system can occur independently of RNA degradation, indicating an underlying mechanism involving translational suppression. This evidence concerns the gene C9orf72 and amyotrophic lateral sclerosis.