Ke et al. designed a gene therapy vector targeting TAR DNA-binding protein (TDP-43) pathology based on the high affinity between 14-3-3θ and the pathogenic TDP-43, significantly alleviating functional deficits and neurodegeneration in different TDP-43 mutant ALS/FTD mouse models [47]. This evidence concerns the gene TARDBP and amyotrophic lateral sclerosis.