Downstream validations of identified hits revealed IGF2BP3 as a specific regulator of CGGexp-RAN translation in different FXTAS cell models, FXTAS patient-derived cells and animal model expressing fragment of mutant FMR1. This multi-domain RNA binding protein can associate with a plethora of targets and influence RNA at different levels (reviewed in ref. 39). The gene discussed is FMR1; the disease is fragile X-associated tremor/ataxia syndrome.