While we currently do not have any data to support this, it is intriguing that Rué et al. observed an improvement of the HD-like phenotype of R6/2 after intrastriatal injection of a CAG-targeting LNA-modified AON, without reduction of HTT protein levels, suggesting that reversal of the detrimental effects of mHTT RNA toxicity may play an important yet so far underappreciated role in HD pathogenesis [25]. Here, HTT is linked to Huntington disease.