Department of Neurology, Kennedy Center for Research on Human Development, Vanderbilt University School of Medicine, Nashville, TN 37232-8552, USA.
Hum Mol Genet. 2013 Jan 1;22(1):140-52. doi: 10.1093/hmg/dds414. Epub 2012 Oct 9.
Tuberous sclerosis complex (TSC) is a multisystem genetic disorder with severe neurologic manifestations, including epilepsy, autism, anxiety and attention deficit hyperactivity disorder. TSC is caused by the loss of either the TSC1 or TSC2 genes that normally regulate the mammalian target of rapamycin (mTOR) kinase. mTOR exists within two distinct complexes, mTOR complex 1 (mTORC1) and mTOR complex 2 (mTORC2). Loss of either TSC gene leads to increased mTORC1 but decreased mTORC2 signaling. As the contribution of decreased mTORC2 signaling to neural development and homeostasis has not been well studied, we generated a conditional knockout (CKO) of Rictor, a key component of mTORC2. mTORC2 signaling is impaired in the brain, whereas mTORC1 signaling is unchanged. Rictor CKO mice have small brains and bodies, normal lifespan and are fertile. Cortical layering is normal, but neurons are smaller than those in control brains. Seizures were not observed, although excessive slow activity was seen on electroencephalography. Rictor CKO mice are hyperactive and have reduced anxiety-like behavior. Finally, there is decreased white matter and increased levels of monoamine neurotransmitters in the cerebral cortex. Loss of mTORC2 signaling in the cortex independent of mTORC1 can disrupt normal brain development and function and may contribute to some of the neurologic manifestations seen in TSC.
结节性硬化症(TSC)是一种多系统遗传疾病,伴有严重的神经系统表现,包括癫痫、自闭症、焦虑和注意力缺陷多动障碍。TSC 是由 TSC1 或 TSC2 基因的缺失引起的,这两个基因通常调节哺乳动物雷帕霉素靶蛋白(mTOR)激酶。mTOR 存在于两个不同的复合物中,即 mTOR 复合物 1(mTORC1)和 mTOR 复合物 2(mTORC2)。任一 TSC 基因的缺失都会导致 mTORC1 信号增加,但 mTORC2 信号减少。由于 mTORC2 信号对神经发育和稳态的贡献尚未得到很好的研究,我们生成了 Rictor(mTORC2 的关键组成部分)的条件敲除(CKO)小鼠。mTORC2 信号在大脑中受损,而 mTORC1 信号不变。Rictor CKO 小鼠的大脑和身体较小,寿命正常且具有生育能力。皮质分层正常,但神经元比对照大脑中的神经元小。虽然脑电图上观察到过度的慢波活动,但未观察到癫痫发作。Rictor CKO 小鼠表现出过度活跃和焦虑样行为减少。最后,大脑皮层的白质减少,单胺神经递质水平增加。mTORC2 信号在皮质中的缺失独立于 mTORC1 可破坏正常的大脑发育和功能,并可能导致 TSC 中一些神经系统表现。