Research into the mechanisms of neurodegeneration in FXTAS has largely focused on how the FMR1 mRNA might elicit a gain of function toxicity, either through sequestration of RNA binding proteins or through triggered aberrant translation through the repeat of aggregate prone proteins that underlie the intranuclear inclusions observed in patients (Jin et al., 2003, 2007; Hagerman and Hagerman, 2004, 2013; Iwahashi et al., 2006; Sofola et al., 2007; Sellier et al., 2010, 2013; Todd and Paulson, 2010; Renoux and Todd, 2012; Todd et al., 2013). Here, FMR1 is linked to fragile X-associated tremor/ataxia syndrome.