Institute of Neuroscience, CNR, 20854, Vedano al Lambro, Italy.
Department of Medical Biotechnology and Translational Medicine, Università degli Studi di Milano, 20129, Milano, Italy.
Mol Psychiatry. 2024 Jun;29(6):1710-1725. doi: 10.1038/s41380-023-02022-1. Epub 2023 Mar 30.
Mutations in PCDH19 gene, which encodes protocadherin-19 (PCDH19), cause Developmental and Epileptic Encephalopathy 9 (DEE9). Heterogeneous loss of PCDH19 expression in neurons is considered a key determinant of the disorder; however, how PCDH19 mosaic expression affects neuronal network activity and circuits is largely unclear. Here, we show that the hippocampus of Pcdh19 mosaic mice is characterized by structural and functional synaptic defects and by the presence of PCDH19-negative hyperexcitable neurons. Furthermore, global reduction of network firing rate and increased neuronal synchronization have been observed in different limbic system areas. Finally, network activity analysis in freely behaving mice revealed a decrease in excitatory/inhibitory ratio and functional hyperconnectivity within the limbic system of Pcdh19 mosaic mice. Altogether, these results indicate that altered PCDH19 expression profoundly affects circuit wiring and functioning, and provide new key to interpret DEE9 pathogenesis.
PCDH19 基因突变,导致发育性和癫痫性脑病 9 型(DEE9)。神经元中 PCDH19 表达的异质性缺失被认为是该疾病的一个关键决定因素;然而,PCDH19 镶嵌表达如何影响神经元网络活动和回路在很大程度上尚不清楚。在这里,我们表明 Pcdh19 镶嵌小鼠的海马体具有结构和功能突触缺陷,以及存在 PCDH19 阴性的过度兴奋神经元。此外,在不同的边缘系统区域观察到网络发射率的整体降低和神经元同步性的增加。最后,在自由活动的小鼠中进行的网络活动分析表明,Pcdh19 镶嵌小鼠的边缘系统中的兴奋性/抑制性比值降低和功能超连接性增加。总的来说,这些结果表明,改变的 PCDH19 表达深刻地影响了电路布线和功能,并为解释 DEE9 的发病机制提供了新的关键。