To validate this activity in a more physiologically relevant model and to assess the impact of targeting this sequence on various C9orf72 transcripts, we retested the activity of a subset of oligonucleotides in C9-ALS iPSC-derived motor neurons (denoted ALS motor neurons, Supplementary Fig. 1d–g). The gene discussed is C9orf72; the disease is amyotrophic lateral sclerosis.