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扩大 HIVEP2 变异引起的智力残疾表型。

Expanding the phenotype of intellectual disability caused by HIVEP2 variants.

机构信息

Hunter Genetics, Waratah, New South Wales, Australia.

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

出版信息

Am J Med Genet A. 2019 Sep;179(9):1872-1877. doi: 10.1002/ajmg.a.61271. Epub 2019 Jun 17.

Abstract

De novo pathogenic variants in the human immunodeficiency virus enhancer type I binding protein 2 (HIVEP2) gene, a large transcription factor predominantly expressed in the brain have previously been associated with intellectual disability (ID) and dysmorphic features in nine patients. We describe the phenotype and genotype of two additional patients with novel de novo pathogenic HIVEP2 variants, who have previously unreported features, including hyperphagia and Angelman-like features. Exome sequencing was utilized in the investigation of the patients who had previously incurred a rigorous genetic workup for their neurodevelopmental delay, and in whom no genetic cause had been detected. Information pertaining to phenotype and genotype for new patients was collated along with data from previous reports, showing that the phenotypic spectrum of patients with HIVEP2 variants is broader than first noted. Additional characteristics are: an increased body mass index; and features of Angelman-like syndromes including: ID, limited speech, post-natal microcephaly, and hypotonia. Dysmorphic features vary between patients. As yet, no clear association between the type of gene aberration and phenotype can be concluded. HIVEP2-related ID needs to be considered in the differential diagnosis of patients with Angelman-like phenotypes and hyperphagia, and whole-exome sequencing should be considered in the genetic diagnostic armamentarium for patients with ID of inconclusive etiology.

摘要

先前已有研究表明,人类免疫缺陷病毒增强子 I 结合蛋白 2(HIVEP2)基因中的新生致病性变异与 9 名患者的智力障碍(ID)和发育异常特征有关,该基因是一种主要在大脑中表达的大型转录因子。我们描述了另外两名具有新的新生致病性 HIVEP2 变异的患者的表型和基因型,他们具有以前未报道过的特征,包括食欲过盛和 Angelman 样特征。对曾因神经发育迟缓进行过严格的遗传研究但未发现遗传病因的患者进行了外显子组测序。我们收集了新患者的表型和基因型信息,并与之前的报告数据进行了汇总,结果表明,HIVEP2 变异患者的表型谱比最初发现的更为广泛。其他特征还包括:体重指数增加;Angelman 样综合征的特征包括:ID、言语有限、出生后小头畸形和低张力。不同患者的发育异常特征不同。目前,还不能得出基因异常类型与表型之间存在明确关联的结论。对于具有 Angelman 样表型和食欲过盛的患者,需要考虑 HIVEP2 相关 ID,并应考虑对病因不明的 ID 患者进行全外显子组测序作为遗传诊断工具。

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