Pathology could occur via poisoning of splicing factors, in particular TDP-43, with non-coding repeats inducing toxic RNA foci that sequester splicing factors and other nuclear proteins (Todd and Paulson, 2010), as in Fragile X Tremor Ataxia Syndrome (FXTAS) and myotonic dystrophy. The gene discussed is TARDBP; the disease is fragile X-associated tremor/ataxia syndrome.