Neurodevelopmental disorders (NDD), including autism spectrum disorder (ASD), are a series of disorders affecting brain development and function and characterized by significant cognitive, emotional, and behavioral symptoms. Gamma-aminobutyric acid (GABA)ergic inhibition plays a critical role in the subtle regulation of neuronal connectivity, morphology, and synaptic transmission throughout early embryonic development to adulthood. A prevailing hypothesis demonstrated that disrupted excitatory/inhibitory(E/I) balance and abnormal synaptic connectivity underlie the pathogenesis of autism. However, the specific molecular mechanism by which GABA and GABAergic synaptic transmission affect NDD remains unclear. In this review, we summarized current evidence focusing on the altered expression and function of the GABAergic system and how it relates to ASD and other ASD -related syndromic disorders and highlighted the new therapeutic perspectives of GABA or GABA modulators for the treatment of ASD.