Targeting Epigenetic Enzymes for Treatment of Autism Spectrum Disorders

Targeting epigenetic enzymes has emerged as a promising therapeutic approach for Autism Spectrum Disorder (ASD). Inhibitors of histone deacetylase (HDAC), such as Romidepsin and MS-275, alongside the EHMT1/2 inhibitor UNC0642 and the LSD1 inhibitor GSK-LSD1, have demonstrated potential in preclinical studies. These compounds have been shown to restore histone acetylation and methylation levels, modulate gene expression, and correct synaptic defects in the prefrontal cortex (PFC), a brain region implicated in ASD pathology. These interventions offer potential pathways for alleviating the behavioral abnormalities associated with ASD. This book chapter underscores the promise of targeting chromatin regulators as therapeutic strategies to improve outcomes for individuals with ASD.