Bedogni Francesco, Cobolli Gigli Clementina, Pozzi Davide, Rossi Riccardo Lorenzo, Scaramuzza Linda, Rossetti Grazisa, Pagani Massimiliano, Kilstrup-Nielsen Charlotte, Matteoli Michela, Landsberger Nicoletta
San Raffaele Rett Research Unit, Division of Neuroscience, San Raffaele Scientific Institute, 20132 Milan, Italy.
Laboratory of Genetic and Epigenetic Control of Gene Expression, Division of Biomedical Research, Department of Theoretical and Applied Sciences, University of Insubria, Busto Arsizio, 21052 Varese, Italy.
Cereb Cortex. 2016 Jun;26(6):2517-2529. doi: 10.1093/cercor/bhv078. Epub 2015 May 15.
MeCP2 is associated with several neurological disorders; of which, Rett syndrome undoubtedly represents the most frequent. Its molecular roles, however, are still unclear, and data from animal models often describe adult, symptomatic stages, while MeCP2 functions during embryonic development remain elusive. We describe the pattern and timing of Mecp2 expression in the embryonic neocortex highlighting its low but consistent expression in virtually all cells and show the unexpected occurrence of transcriptional defects in the Mecp2 null samples at a stage largely preceding the onset of overt symptoms. Through the deregulated expression of ionic channels and glutamatergic receptors, the lack of Mecp2 during early neuronal maturation leads to the reduction in the neuronal responsiveness to stimuli. We suggest that such features concur to morphological alterations that begin affecting Mecp2 null neurons around the perinatal age and become evident later in adulthood. We indicate MeCP2 as a key modulator of the transcriptional mechanisms regulating cerebral cortex development. Neurological phenotypes of MECP2 patients could thus be the cumulative result of different adverse events that are already present at stages when no obvious signs of the pathology are evident and are worsened by later impairments affecting the central nervous system during maturation and maintenance of its functionality.
MeCP2与多种神经疾病相关;其中,雷特综合征无疑最为常见。然而,其分子作用仍不清楚,动物模型的数据通常描述的是成年有症状阶段,而MeCP2在胚胎发育过程中的功能仍不清楚。我们描述了Mecp2在胚胎新皮层中的表达模式和时间,突出了其在几乎所有细胞中低但一致的表达,并显示在明显症状出现之前的一个阶段,Mecp2基因敲除样本中意外出现转录缺陷。在早期神经元成熟过程中,由于离子通道和谷氨酸能受体的表达失调,Mecp2的缺失导致神经元对刺激的反应性降低。我们认为,这些特征共同导致形态学改变,这种改变在围产期开始影响Mecp2基因敲除的神经元,并在成年后期变得明显。我们指出MeCP2是调节大脑皮层发育的转录机制的关键调节因子。因此,MECP2患者的神经表型可能是不同不良事件的累积结果,这些不良事件在病理无明显迹象的阶段就已存在,并在中枢神经系统成熟和维持其功能期间受到后期损伤的影响而恶化。