Do we have a cure for tuberous sclerosis complex?
Do we have a cure for tuberous sclerosis complex?
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DOI：
10.1111/j.1535-7511.2008.00279.x
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发表时间：
2008-11-01
影响因子：
3.6
通讯作者：
Crino, Peter B
中科院分区：
文献类型：
--
作者：
Crino, Peter B
最近几种结节性硬化症（TSC）小鼠模型的发展为测试TSC神经系统表现的新疗法提供了体内系统。在体外和小鼠TSC模型中，雷帕霉素可以拮抗TSC蛋白功能丧失的影响，雷帕霉素可以预防癫痫发作和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提供了新的希望。
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.