Indeed, we found that small hairpin RNA (shRNA)-mediated ATXN3 knockdown (Supplementary Fig. 12) results in the substantial accumulation and hyperphosphorylation of TDP-43 CTFs at S409/S410 in HEK293T cells (Fig. 5g, i, j) and SH-SY5Y neuroblastoma cells (Fig. 5h–l). The gene discussed is ATXN3; the disease is neuroblastoma.