Department of Human Genetics, University of Utah School of Medicine, Salt Lake City, Utah, United States of America.
Department of Biological Sciences, University of Pittsburgh, Pittsburgh, Pennsylvania, United States of America.
PLoS Genet. 2024 Sep 11;20(9):e1011406. doi: 10.1371/journal.pgen.1011406. eCollection 2024 Sep.
Mutations in glycosylation pathways, such as N-linked glycosylation, O-linked glycosylation, and GPI anchor synthesis, lead to Congenital Disorders of Glycosylation (CDG). CDG typically present with seizures, hypotonia, and developmental delay but display large clinical variability with symptoms affecting every system in the body. This variability suggests modifier genes might influence the phenotypes. Because of the similar physiology and clinical symptoms, there are likely common genetic modifiers between CDG. Here, we use evolution as a tool to identify common modifiers between CDG and glycosylation genes. Protein glycosylation is evolutionarily conserved from yeast to mammals. Evolutionary rate covariation (ERC) identifies proteins with similar evolutionary rates that indicate shared biological functions and pathways. Using ERC, we identified strong evolutionary rate signatures between proteins in the same and different glycosylation pathways. Genome-wide analysis of proteins showing significant ERC with GPI anchor synthesis proteins revealed strong signatures with ncRNA modification proteins and DNA repair proteins. We also identified strong patterns of ERC based on cellular sub-localization of the GPI anchor synthesis enzymes. Functional testing of the highest scoring candidates validated genetic interactions and identified novel genetic modifiers of CDG genes. ERC analysis of disease genes and biological pathways allows for rapid prioritization of potential genetic modifiers, which can provide a better understanding of disease pathophysiology and novel therapeutic targets.
糖基化途径中的突变,如 N 连接糖基化、O 连接糖基化和 GPI 锚合成,导致先天性糖基化障碍 (CDG)。CDG 通常表现为癫痫、张力减退和发育迟缓,但表现出很大的临床变异性,症状影响身体的每个系统。这种变异性表明修饰基因可能影响表型。由于相似的生理学和临床症状,CDG 之间可能存在共同的遗传修饰因子。在这里,我们利用进化作为一种工具来识别 CDG 和糖基化基因之间的共同修饰因子。蛋白质糖基化在从酵母到哺乳动物的进化过程中是保守的。进化率协变 (ERC) 识别具有相似进化率的蛋白质,这些蛋白质表明具有共同的生物学功能和途径。利用 ERC,我们在同一和不同糖基化途径中的蛋白质之间鉴定出了强烈的进化率特征。对与 GPI 锚合成蛋白显示出显著 ERC 的蛋白进行全基因组分析,揭示了与 ncRNA 修饰蛋白和 DNA 修复蛋白的强烈特征。我们还根据 GPI 锚合成酶的细胞亚定位鉴定了基于 ERC 的强烈模式。对得分最高的候选物进行功能测试验证了遗传相互作用,并确定了 CDG 基因的新遗传修饰因子。对疾病基因和生物途径进行 ERC 分析可以快速确定潜在的遗传修饰因子,从而更好地理解疾病的病理生理学和新的治疗靶点。