The recent development of several mouse models for tuberous sclerosis complex (TSC) provides in vivo systems to test new therapies for the neurological manifestations of TSC. Rapamycin is known to antagonize the effects of loss of TSC protein function in vitro and in mouse TSC models, rapamycin can prevent seizures and improve learning task performance. These findings provide new hope for TSC patients suffering from intractable seizures and possibly, for those with autism and cognitive disabilities.

中文翻译：

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我们有治愈结节性硬化症的方法吗？

最近开发的几种结节性硬化症 (TSC) 小鼠模型提供了体内系统来测试 TSC 神经学表现的新疗法。众所周知，雷帕霉素在体外和小鼠 TSC 模型中可以拮抗 TSC 蛋白功能丧失的影响，雷帕霉素可以预防癫痫发作并提高学习任务的表现。这些发现为患有顽固性癫痫发作的 TSC 患者以及可能患有自闭症和认知障碍的患者带来了新的希望。