Previous studies have shown that iPSC-derived motor neurons bearing ALS relevant TARDBP mutations exhibit significant levels of translocation of the TDP-43 protein from the normal nuclear location to the cytoplasm, associated with disruption of nuclear RNA splicing functions and with the formation of potentially toxic cytoplasmic aggregates (Winton et al., 2008; Zhang et al., 2009). The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.