Indeed, potential therapeutics for ALS (e.g. ataxin 2 antisense oligonucleotide, ION541/BIIB105) and PD (e.g. stearoyl-CoA-desaturase inhibitor, YTX-7739) that have emerged from these studies are now entering phase 1 clinical trials. The gene discussed is ATXN2; the disease is amyotrophic lateral sclerosis.