C9orf72 and amyotrophic lateral sclerosis: The researchers used 2′-MOE P = S gapmer ASO to target exon 2 shared by the c9orf72 transcript and the intron sequence near the repetitive sequence to degrade target mRNA, thereby reducing pathological RNA foci of fibroblasts and inducing pluripotent stem cells from C9ORF72 ALS patient [75, 76].