研究人员第一次在动物模型中，抑制了发生于脑部的癫痫。他们利用基因疗法修改脑部的信号路径，发现可以显著地减少大鼠的癫痫症发作。
这项研究是由宾州大学医学院小儿科神经学家Amy R. Brooks-Kayal所引导。他表示，这项研究提供了一个概念，证明可经由修改脑部神经细胞中的特殊信号路径，来治疗癫痫症。这项研究结果发表于11月1 日的Journal of Neuroscience中。
研究人员进行治疗的脑部区域称为齿状回（dentate gyrus），他们把焦点集中于一种gamma-aminobutyric acid (GABA)A型受体。当GABA(A)受体被活化后，可抑制脑细胞反复过度的刺激所引发的癫痫。
癫痫是由于刺激神经元的二种神经传导物质：谷胺酸与GABA系统的不平衡所致。GABA所扮演的抑制角色在齿状回中是特别重要的，因为齿状回是脑部的活动进入海马回的门户，而海马回是引起最常见的儿童和成人癫痫类型：颞叶癫痫之部位。GABA(A)受体含有五个次单位，研究人员之前在动物研究中，发现有癫痫的大鼠之alpha1次单位含量偏低且alpha4 次单位含量偏高。
所以，研究人员传输基因以修改alpha1 次单位的表现，结果发现可以使alpha1 蛋白质的量增加，而使癫痫的发生率减少了三倍。虽然这只是一项短期研究，无法预测长期的结果，但却有助于引导出治疗癫痫的基因疗法。
部分英文原文：
Enhancing GABAA Receptor 1 Subunit Levels in Hippocampal Dentate Gyrus Inhibits Epilepsy Development in an Animal Model of Temporal Lobe Epilepsy.
Differential expression of GABA(A) receptor (GABR) subunits has been demonstrated in hippocampus from patients and animals with temporal lobe epilepsy (TLE), but whether these changes are important for epileptogenesis remains unknown. Previous studies in the adult rat pilocarpine model of TLE found reduced expression of GABR alpha1 subunits and increased expression of alpha4 subunits in dentate gyrus (DG) of epileptic rats compared with controls. To investigate whether this altered subunit expression is a critical determinant of spontaneous seizure development, we used adeno-associated virus type 2 containing the alpha4 subunit gene (GABRA4) promoter to drive transgene expression in DG after status epilepticus (SE). This novel use of a condition-dependent promoter upregulated after SE successfully increased expression of GABR alpha1 subunit mRNA and protein in DG at 1-2 weeks after SE. Enhanced alpha1 expression in DG resulted in a threefold increase in mean seizure-free time after SE and a 60% decrease in the number of rats developing epilepsy (recurrent spontaneous seizures) in the first 4 weeks after SE. These findings provide the first direct evidence that altering GABR subunit expression can affect the development of epilepsy and suggest that alpha1 subunit levels are important determinants of inhibitory function in hippocampus.
