Figure 3. Toward a therapy for FXS.
(A) Epigenetic-modifying compounds that may reactivate FMR1 gene expression. (B) Compounds used in clinical trials and affecting dysregulated molecules and pathways are shown. Lack of FMRP leads to a dysregulated synthesis of molecules with key functions at synapses, such as increased levels of Arc, αCAMKII, MMP9, GSK-3β, mGluR, and NMDA and decreased GABA and AMPA receptor activity. Arrows and inhibition symbols do not necessarily represent direct interactions.