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One of the first approaches that was suggested for FXS treatment was the Fmr1 gene activity restoration through changes in the DNA methylation levels and epigenetic modifications ( in vitro reactivation of the Fmr1 gene, such as 5-azadeoxycytidine (5-azadC) ( in vivo studies due to safety problems related to low reactivity and high toxicity of these chromatin-modifying enzymes inhibitors ( Fmr1 gene ( Fmr1 mRNA with the full mutation could be toxic, as it was demonstrated by a correlation detected between the Fmr1 mRNA levels in blood and more severe autism features ( However, since several compounds are used to treat behavioral and mental problems, such as stimulants or antipsychotics ( Fmr1 KO mice these agents showed improvement of several FXS features, including better behavioral abilities, restoration of normal levels of dendritic spines and reduction in protein synthesis ( Most of drugs tested in FXS pharmacotherapy are compounds already employed or approved for other disorders

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Rymaszewski, Z., Cohen, R