Crosstalk between serotonin and mTOR systems: molecular pathology in autism and its treatment

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We conducted a translational research to understand the pathomechanism of autism on the basis of abnormal signal transduction in the brain, and to establish animal models for the development of drug treatment (molecular target therapy). Using animal models (Tsc1 and Tsc2 knockout mice) of tuberous sclerosis complex (TSC), an autosomal dominantly inherited disorder that frequently presents with autism, we observed the cardinal symptom of autism, impaired social interaction, and its improvement by treatment with an mTOR inhibitor, elucidated the molecular basis, and recapitulated the clinical findings in human TSC patients that Tsc2 mutations tend to be severer than Tsc1 mutations.