Tranchevent Léon-Charles, Halder Rashi, Glaab Enrico
Luxembourg Centre for Systems Biomedicine (LCSB), University of Luxembourg, Esch-sur-Alzette, Luxembourg.
NPJ Parkinsons Dis. 2023 Jan 21;9(1):8. doi: 10.1038/s41531-023-00446-8.
Parkinson's disease (PD) is a heterogeneous disorder, and among the factors which influence the symptom profile, biological sex has been reported to play a significant role. While males have a higher age-adjusted disease incidence and are more frequently affected by muscle rigidity, females present more often with disabling tremors. The molecular mechanisms involved in these differences are still largely unknown, and an improved understanding of the relevant factors may open new avenues for pharmacological disease modification. To help address this challenge, we conducted a meta-analysis of disease-associated molecular sex differences in brain transcriptomics data from case/control studies. Both sex-specific (alteration in only one sex) and sex-dimorphic changes (changes in both sexes, but with opposite direction) were identified. Using further systems level pathway and network analyses, coordinated sex-related alterations were studied. These analyses revealed significant disease-associated sex differences in mitochondrial pathways and highlight specific regulatory factors whose activity changes can explain downstream network alterations, propagated through gene regulatory cascades. Single-cell expression data analyses confirmed the main pathway-level changes observed in bulk transcriptomics data. Overall, our analyses revealed significant sex disparities in PD-associated transcriptomic changes, resulting in coordinated modulations of molecular processes. Among the regulatory factors involved, NR4A2 has already been reported to harbor rare mutations in familial PD and its pharmacological activation confers neuroprotective effects in toxin-induced models of Parkinsonism. Our observations suggest that NR4A2 may warrant further research as a potential adjuvant therapeutic target to address a subset of pathological molecular features of PD that display sex-associated profiles.
帕金森病(PD)是一种异质性疾病,在影响症状表现的因素中,据报道生物性别起着重要作用。虽然男性经年龄调整后的疾病发病率较高,且更常受到肌肉僵硬的影响,但女性更常出现致残性震颤。这些差异所涉及的分子机制在很大程度上仍不清楚,更好地了解相关因素可能会为疾病的药物修饰开辟新途径。为了帮助应对这一挑战,我们对病例/对照研究的脑转录组学数据中与疾病相关的分子性别差异进行了荟萃分析。我们识别出了性别特异性(仅在一种性别中发生改变)和性别二态性变化(在两种性别中均发生变化,但方向相反)。通过进一步的系统水平通路和网络分析,研究了与性别相关的协同改变。这些分析揭示了线粒体通路中与疾病相关的显著性别差异,并突出了特定的调节因子,其活性变化可以解释通过基因调控级联传播的下游网络改变。单细胞表达数据分析证实了在整体转录组学数据中观察到的主要通路水平变化。总体而言,我们的分析揭示了PD相关转录组变化中存在显著的性别差异,从而导致分子过程的协同调节。在涉及的调节因子中,NR4A2已被报道在家族性PD中存在罕见突变,并且其药理学激活在帕金森病毒素诱导模型中具有神经保护作用。我们的观察结果表明,NR4A2作为一个潜在的辅助治疗靶点,可能值得进一步研究,以解决PD中一部分表现出性别相关特征的病理分子特征。