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Utility of spontaneous animal models of Alzheimers disease in preclinical efficacy studies

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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

Multiple accounts describe reduced pain and improved mobility by day 3, often accompanied by disbelief that effects could appear so quickly