Genetic disorders that present with a high incidence of autism spectrum 
disorders (ASD) offer tremendous potential both for elucidating the underlying 
neurobiology of ASD and identifying therapeutic drugs and/or drug targets. As a 
result, clinical trials for genetic disorders associated with ASD are no longer 
a hope for the future but rather an exciting reality whose time has come. 
Tuberous sclerosis complex (TSC) is one such genetic disorder that presents with 
ASD, epilepsy, and intellectual disability. Cell culture and mouse model 
experiments have identified the mTOR pathway as a therapeutic target in this 
disease. This review summarizes the advantages of using TSC as model of ASD and 
the recent advances in the translational and clinical treatment trials in TSC.