C9orf72 and amyotrophic lateral sclerosis: These data sets were: (i) proteome of C9orf72 HRE RBP, which provides the most important biochemical information of C9-FTD/ALS (Cooper-Knock et al., 2014b; Haeusler et al., 2014); (ii) transcriptome of iPSNs of patients with C9-ALS (Sareen et al., 2013); and (iii) transcriptome of motor neurons of C9-FTD/ALS patients acting as the most clinically appropriate in vivo source (Highley et al., 2014).