Department of Cognitive Neuroscience, Donders Institute for Brain Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, The Netherlands.
Department of Human Genetics, Radboud University Medical Center, 6525 GA Nijmegen, The Netherlands.
Int J Mol Sci. 2023 Jan 11;24(2):1428. doi: 10.3390/ijms24021428.
Tourette's disorder (TD) is a highly heritable childhood-onset neurodevelopmental disorder and is caused by a complex interplay of multiple genetic and environmental factors. Yet, the molecular mechanisms underlying the disorder remain largely elusive. In this study, we used the available omics data to compile a list of TD candidate genes, and we subsequently conducted tissue/cell type specificity and functional enrichment analyses of this list. Using genomic data, we also investigated genetic sharing between TD and blood and cerebrospinal fluid (CSF) metabolite levels. Lastly, we built a molecular landscape of TD through integrating the results from these analyses with an extensive literature search to identify the interactions between the TD candidate genes/proteins and metabolites. We found evidence for an enriched expression of the TD candidate genes in four brain regions and the pituitary. The functional enrichment analyses implicated two pathways ('cAMP-mediated signaling' and 'Endocannabinoid Neuronal Synapse Pathway') and multiple biological functions related to brain development and synaptic transmission in TD etiology. Furthermore, we found genetic sharing between TD and the blood and CSF levels of 39 metabolites. The landscape of TD not only provides insights into the (altered) molecular processes that underlie the disease but, through the identification of potential drug targets (such as FLT3, NAALAD2, CX3CL1-CX3CR1, OPRM1, and HRH2), it also yields clues for developing novel TD treatments.
妥瑞氏症(TD)是一种高度遗传性的儿童期发病的神经发育障碍,是由多种遗传和环境因素的复杂相互作用引起的。然而,该疾病的分子机制在很大程度上仍难以捉摸。在这项研究中,我们使用现有的组学数据编制了一份 TD 候选基因列表,然后对该列表进行了组织/细胞类型特异性和功能富集分析。我们还使用基因组数据研究了 TD 与血液和脑脊液(CSF)代谢物水平之间的遗传共享。最后,我们通过将这些分析的结果与广泛的文献搜索相结合,构建了一个 TD 的分子图谱,以识别 TD 候选基因/蛋白与代谢物之间的相互作用。我们发现 TD 候选基因在四个大脑区域和脑垂体中有丰富的表达。功能富集分析表明,在 TD 发病机制中存在两种途径(“cAMP 介导的信号转导”和“内源性大麻素神经元突触途径”)和多个与大脑发育和突触传递相关的生物学功能。此外,我们发现 TD 与血液和 CSF 中 39 种代谢物的水平之间存在遗传共享。TD 的分子图谱不仅提供了对潜在疾病分子过程的深入了解,而且通过鉴定潜在的药物靶点(如 FLT3、NAALAD2、CX3CL1-CX3CR1、OPRM1 和 HRH2),为开发新的 TD 治疗方法提供了线索。