A causal relationship has been established between disturbed activation of mTORC1 signaling pathway and several neurological abnormalities observed in different well-characterized monogenic syndromes with high prevalence of ASD (TSC, PTEN-ASD, FXS, AS and RTT), and preclinical studies have shown that modulation of mTORC1 signaling may provide promising avenues for the treatment of ASD-relevant symptoms. The gene discussed is TSC1; the disease is Rett syndrome.