Behavioral pharmacological study to develop animal model systems for creating drugs to treat autism

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Drug therapy that effectively improves social interaction, a core symptom of autism, is awaited. Tuberous sclerosis complex(TSC) is caused by haploinsufficiency of the TSC1 or TSC2 gene, and is often manifested with autism. This study found in animal models of TSC, Tsc1^<+/-> and Tsc2^<+/-> mice, distinct autism-like behavioral features, and their recovery, as well as normalization of expression of multiple genes involved in mammalian target of rapamycin(mTOR) signaling, after treatment with an mTOR inhibitor, rapamycin. These findings indicate the crucial role of mTOR signaling in autism-like behaviors in mice, and suggests the usefulness of mTOR inhibitors in the pharmacological treatment of human autism.