Using the ALS-iPSCs system, it was shown that all toxic phenotypes were alleviated by treatment of induced pluripotent stem cell-derived neurons (iPSNs) with novel antisense oligonucleotides (ASOs) targeting GGGGCCexp RNA sequences, while C9ORF72 RNA levels were not reduced. The gene discussed is C9orf72; the disease is amyotrophic lateral sclerosis.