Manchester Centre for Genomic Medicine, Division of Evolution & Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester M13 9WL, UK; Laboratorio de Genética y Enfermedades Metabólicas, Instituto de Nutrición y Tecnología de los Alimentos , Universidad de Chile, Santiago 7830490, Chile.
Manchester Centre for Genomic Medicine, Division of Evolution & Genomic Sciences, School of Biological Sciences, Faculty of Biology, Medicine and Health, University of Manchester, Manchester M13 9WL, UK; Manchester Centre for Genomic Medicine, St Mary's Hospital, Manchester University NHS Foundation Trust, Health Innovation Manchester, Manchester M13 9WL, UK.
Am J Hum Genet. 2018 Jan 4;102(1):175-187. doi: 10.1016/j.ajhg.2017.11.013. Epub 2017 Dec 21.
Histone lysine methyltransferases (KMTs) and demethylases (KDMs) underpin gene regulation. Here we demonstrate that variants causing haploinsufficiency of KMTs and KDMs are frequently encountered in individuals with developmental disorders. Using a combination of human variation databases and existing animal models, we determine 22 KMTs and KDMs as additional candidates for dominantly inherited developmental disorders. We show that KMTs and KDMs that are associated with, or are candidates for, dominant developmental disorders tend to have a higher level of transcription, longer canonical transcripts, more interactors, and a higher number and more types of post-translational modifications than other KMT and KDMs. We provide evidence to firmly associate KMT2C, ASH1L, and KMT5B haploinsufficiency with dominant developmental disorders. Whereas KMT2C or ASH1L haploinsufficiency results in a predominantly neurodevelopmental phenotype with occasional physical anomalies, KMT5B mutations cause an overgrowth syndrome with intellectual disability. We further expand the phenotypic spectrum of KMT2B-related disorders and show that some individuals can have severe developmental delay without dystonia at least until mid-childhood. Additionally, we describe a recessive histone lysine-methylation defect caused by homozygous or compound heterozygous KDM5B variants and resulting in a recognizable syndrome with developmental delay, facial dysmorphism, and camptodactyly. Collectively, these results emphasize the significance of histone lysine methylation in normal human development and the importance of this process in human developmental disorders. Our results demonstrate that systematic clinically oriented pathway-based analysis of genomic data can accelerate the discovery of rare genetic disorders.
组蛋白赖氨酸甲基转移酶(KMTs)和去甲基化酶(KDMs)是基因调控的基础。在这里,我们证明导致 KMTs 和 KDMs 单倍不足的变体在发育障碍患者中经常遇到。我们使用人类变异数据库和现有的动物模型的组合,确定了 22 种 KMTs 和 KDMs 是另外的显性遗传性发育障碍候选基因。我们表明,与显性发育障碍相关的 KMTs 和 KDMs 或它们的候选基因往往具有更高的转录水平、更长的经典转录本、更多的相互作用因子、更多和更多类型的翻译后修饰,而其他 KMT 和 KDMs 则没有。我们提供了确凿的证据,将 KMT2C、ASH1L 和 KMT5B 的单倍不足与显性发育障碍联系起来。KMT2C 或 ASH1L 的单倍不足导致主要是神经发育障碍,偶尔伴有身体异常,而 KMT5B 突变导致过度生长综合征伴智力障碍。我们进一步扩展了 KMT2B 相关疾病的表型谱,并表明一些个体在至少到儿童中期之前可能存在严重的发育迟缓而没有肌张力障碍。此外,我们描述了一种由同源或复合杂合 KDM5B 变体引起的隐性组蛋白赖氨酸甲基化缺陷,导致具有发育迟缓、面部畸形和爪形手的可识别综合征。总的来说,这些结果强调了组蛋白赖氨酸甲基化在正常人类发育中的重要性,以及该过程在人类发育障碍中的重要性。我们的结果表明,系统地进行基于临床的靶向分析基因组数据可以加速罕见遗传疾病的发现。
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