Although this mouse model has exhibited no survival or motor deficits (42), the mice still express C9orf72 DPRs that can be used as a pathological marker for C9orf72-associated ALS/FTD (4, 42), allowing us to use it to test the efficiency of our CRISPR/Cas13d system in vivo. This evidence concerns the gene C9orf72 and amyotrophic lateral sclerosis.