Notably, an antisense oligonucleotide (ASO) directed against ataxin-2 was found to lower the toxicity of TDP-43 in a transgenic mouse model of the pathology26, with a related approach now under evaluation in a clinical trial for ALS (ClinicalTrials.gov Identifier: NCT04494256). Here, TARDBP is linked to amyotrophic lateral sclerosis.