After treatment of C9FTD/ALS cells in in vitro [54, 69, 71] and in vivo models with ASOs (mice expressing bacterial artificial chromosome (BAC) with the human expanded C9orf72 gene [91] and adeno-associated virus (AAV) (G4C2)66 mice [133]), a reduction in RNA foci was observed. The gene discussed is C9orf72; the disease is amyotrophic lateral sclerosis.