On the one hand, in neurons, TDP-43 is able to shift to the cytoplasm and travel along the axons to bring mRNA to the synapses for local translation, a function notably impaired in stem-cell-derived motor neurons from ALS patients bearing TDP-43 ALS-causing mutations [25,26,27,28]. The gene discussed is TARDBP; the disease is amyotrophic lateral sclerosis.