Motor neuron degeneration due to TDP-43 aggregation pathology cannot be rescued by knockdown of TDP-43 itself, in view of the embryonal lethality of TDP-43 mutants, but the knockdown of the ALS risk factor ataxin-2 has been successful in preventing the neurodegenerative process [49]. This evidence concerns the gene TARDBP and amyotrophic lateral sclerosis.