At the same time, many questions remain to address, including the efficacy and long-term effects of SOD1 ASO, if there could be a treatment for C9 ALS/FTD, FUS ALS and if restoring stathmin-2 or UNC13A function using gene therapy or antisense oligos targeting unwanted cryptic slicing events or rescuing the function of TDP-43 in the nucleus or targeting ATXN2 could be beneficial for ALS patients. The gene discussed is UNC13A; the disease is amyotrophic lateral sclerosis.