Originally identified for its involvement in RNA processing, TDP-43 has emerged as a key player in neurodegeneration, with its aggregation and mis-localization being recognized as prominent pathological hallmarks in ALS and other related disorders such as frontotemporal lobar degeneration (FTLD), limbic-predominant age-related TDP-43 encephalopathy (LATE) [2], and Alzheimer’s disease [3,4,5]. This evidence concerns the gene TARDBP and amyotrophic lateral sclerosis.