Finally, we describe the application of innovative technologies to target directly the FMR1-expanded region in FXS by Clustered Regularly Interspaced Short Palindromic Repeats/Cas9 (CRISPR/Cas9) methodologies, or indirectly through the correction of protein insufficiency in FRDA by synthetic long non-coding RNAs named Short interspersed nuclear element-containing translation UP-regulators (SINEUPs). The gene discussed is FMR1; the disease is fragile X syndrome.