Université de Strasbourg, CNRS, INSERM, Institut de Génétique et de Biologie Moléculaire et Cellulaire (IGBMC), department of translational medicine and neurogenetics 1 rue Laurent Fries, 67404 Illkirch Graffenstaden, France.
Université de Strasbourg, CNRS, INSERM, Institut Clinique de la Souris (ICS), CELPHEDIA, PHENOMIN, 1 rue Laurent Fries, 67404 Illkirch Graffenstaden, France.
Hum Mol Genet. 2021 May 28;30(9):771-788. doi: 10.1093/hmg/ddab012.
Down syndrome (DS) is the most common genetic form of intellectual disability caused by the presence of an additional copy of human chromosome 21 (Hsa21). To provide novel insights into genotype-phenotype correlations, we used standardized behavioural tests, magnetic resonance imaging and hippocampal gene expression to screen several DS mouse models for the mouse chromosome 16 region homologous to Hsa21. First, we unravelled several genetic interactions between different regions of chromosome 16 and how they contribute significantly to altering the outcome of the phenotypes in brain cognition, function and structure. Then, in-depth analysis of misregulated expressed genes involved in synaptic dysfunction highlighted six biological cascades centred around DYRK1A, GSK3β, NPY, SNARE, RHOA and NPAS4. Finally, we provide a novel vision of the existing altered gene-gene crosstalk and molecular mechanisms targeting specific hubs in DS models that should become central to better understanding of DS and improving the development of therapies.
唐氏综合征(DS)是最常见的由人类 21 号染色体(Hsa21)额外拷贝引起的遗传性智力障碍。为了深入了解基因型-表型相关性,我们使用标准化的行为测试、磁共振成像和海马基因表达,筛选了几种与 Hsa21 同源的 16 号染色体的 DS 小鼠模型。首先,我们揭示了 16 号染色体不同区域之间的几种遗传相互作用,以及它们如何显著改变大脑认知、功能和结构表型的结果。然后,对涉及突触功能障碍的失调表达基因的深入分析突出了六个以 DYRK1A、GSK3β、NPY、SNARE、RHOA 和 NPAS4 为中心的生物学级联反应。最后,我们为 DS 模型中特定枢纽的现有改变基因-基因相互作用和分子机制提供了新的视角,这应该成为更好地理解 DS 和改善治疗方法的核心。