Abstract: Autism spectrum disorder (ASD) is becoming one of the fastest growing neurodevelopmen-tal disorders around the world, yet its clinical treatment still faces challenge due to the heterogeneity in etiology and symptom phenotypes. It is believed that excitatory-inhibitory (E-I) imbalance in the central nervous system may play an important role in the pathogenic mechanisms of ASD. E-I imbalances in synaptic transmission and neural circuits are frequently observed in different animal models of ASD, and the corresponding reversion normalizes the autism-like behaviors in these animals. Some E-I modulators have been tested for their therapeutic potential on ASD patients with encouraging results. This article expounds the mechanism of E-I imbalance in ASD and E-I imbalance regulators treatment progress, to provide new insights on the therapeutic targets for ASD.