Moreover, CRISPR-Cas9-mediated loss of AFF2/FMR2, one of four human homologs of Lilli, specifically decreased the levels of C9ORF72 RNA, RNA foci, and DPR proteins in neurons derived from iPSCs of patients with C9ORF72-ALS/FTD. Here, C9orf72 is linked to amyotrophic lateral sclerosis.