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新发现的 TRIP12 基因新生突变揭示了可变的表型表现,同时强调了 TRIP12 变异的核心特征。

Novel de novo TRIP12 mutation reveals variable phenotypic presentation while emphasizing core features of TRIP12 variations.

机构信息

University of Newcastle, Callaghan, New South Wales, Australia.

Department of Molecular Medicine, John Hunter Hospital, New Lambton Heights, New South Wales, Australia.

出版信息

Am J Med Genet A. 2020 Jul;182(7):1801-1806. doi: 10.1002/ajmg.a.61618. Epub 2020 May 19.

Abstract

Intellectual disability (ID) is a complicated and multifactorial condition often with an unclear cause. Advancements in diagnostic techniques have identified genetic causes in a significant proportion. Pathogenic variants in TRIP12, encoding for an E3 ligand in the ubiquitin-protease pathway, have previously been identified as a cause of ID with autistic behavior and dysmorphic features. We report two unrelated patients with de novo mutations in TRIP12 and diagnoses of global developmental delay, autism spectrum disorder and dysmorphic features, as well as a range of other characteristics. Exome sequencing was utilized as part of an extensive genetic workup for both individuals. The genotypic and phenotypic data for both patients has been collated with previously reported data. Epilepsy was noted in about 20% published cases. One of our patents had epilepsy. These cases highlight the variable phenotypic presentations of TRIP12 variations while emphasizing the core features of ID and speech delay, with or without autistic features and epilepsy.

摘要

智力障碍(ID)是一种复杂的多因素疾病,通常病因不明。诊断技术的进步已经确定了很大一部分遗传原因。先前已发现编码泛素蛋白酶途径 E3 配体的 TRIP12 中的致病性变异是具有自闭症行为和发育异常特征的 ID 的原因。我们报告了两名无关的患者,他们在 TRIP12 中存在新生突变,并被诊断为全面发育迟缓、自闭症谱系障碍和发育异常,以及一系列其他特征。外显子组测序被用作两个人的广泛遗传研究的一部分。两名患者的基因型和表型数据与之前报道的数据进行了整理。大约 20%的已发表病例中存在癫痫。我们的一位患者有癫痫。这些病例突出了 TRIP12 变异的可变表型表现,同时强调了 ID 和言语延迟的核心特征,无论是否伴有自闭症特征和癫痫。

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