VNG1.47 and IGS2.7 have shown to be effective in the experimental treatment of lymphoblasts from ALS patients which are characterized by presenting TDP-43 hyperphosphorylated cytosolic aggregates, the main pathological hallmark of ALS (Martinez-Gonzalez et al., 2020; Nozal et al., 2022). Here, TARDBP is linked to amyotrophic lateral sclerosis.