Interestingly, all these genes are involved in brain functions/neurodegenerative pathways and IGF2 has already been identified as a protective factor in oculomotor neurons of ALS patients.35 However, a particularly novel finding of our approach has been the identification of Nitric Oxide Synthase 1 Adaptor Protein (NOS1AP, alias CAPON) as a novel and most promising target capable of rescuing TDP-43 pathology in neuronal alterations. Here, TARDBP is linked to amyotrophic lateral sclerosis.