Department of Neuroscience, Psychology, Drug Research and Child Health NEUROFARBA, University of Florence, Florence, Italy.
Dev Neurobiol. 2014 Feb;74(2):178-96. doi: 10.1002/dneu.22114. Epub 2013 Oct 21.
Rett syndrome (RTT) is a disorder that is caused in the majority of cases by mutations in the gene methyl-CpG-binding protein-2 (MeCP2). Children with RTT are generally characterized by normal development up to the first year and a half of age, after which they undergo a rapid regression marked by a deceleration of head growth, the onset of stereotyped hand movements, irregular breathing, and seizures. Animal models of RTT with good construct and face validity are available. Their analysis showed that homeostatic regulation of MeCP2 gene is necessary for normal CNS functioning and that multiple complex pathways involving different neuronal and glial cell types are disrupted in RTT models. However, it is increasingly clear that RTT pathogenetic mechanisms converge at synaptic level impairing synaptic transmission and plasticity. We review novel findings showing how specific synaptic mechanisms and related signaling pathways are affected in RTT models.
雷特综合征(RTT)是一种疾病,在大多数情况下是由甲基化CpG 结合蛋白 2 (MeCP2)基因的突变引起的。患有 RTT 的儿童通常在一岁半之前发育正常,之后会出现快速退化,表现为头围增长减缓、出现刻板的手部动作、呼吸不规则和癫痫发作。具有良好构建和表面效度的 RTT 动物模型是可用的。它们的分析表明,MeCP2 基因的内稳态调节对于中枢神经系统的正常功能是必要的,并且涉及不同神经元和神经胶质细胞类型的多个复杂途径在 RTT 模型中被破坏。然而,越来越清楚的是,RTT 的发病机制在突触水平上汇聚,损害了突触传递和可塑性。我们综述了新的发现,这些发现表明了特定的突触机制和相关的信号通路在 RTT 模型中是如何受到影响的。