Tuberous sclerosis, which is a monogenic disease and causing cystic lesions and angiomyolipomas in kidneys as well as other multisystemic abnormalities, mutations in TSC1 or TSC2 leads hyperactivation of mTOR and mTOR inhibitors might be the first effective treatment for these patients [22,28]. The gene discussed is TSC1; the disease is tuberous sclerosis.