Suppr超能文献

患者携带 FBXO11 基因新型致病变异的新眼部发现。

New ocular findings in a patient with a novel pathogenic variant in the FBXO11 gene.

机构信息

Serviço de Genética Médica, Departamento de Pediatria, Hospital de Santa Maria, Centro Hospitalar Universitário Lisboa Norte, Lisbon, Portugal.

Serviço de Genética Médica, Departamento de Pediatria, Hospital de Santa Maria, Centro Hospitalar Universitário Lisboa Norte, Lisbon, Portugal.

出版信息

J AAPOS. 2022 Oct;26(5):268-270. doi: 10.1016/j.jaapos.2022.05.008. Epub 2022 Aug 5.

Abstract

Intellectual developmental disorder with dysmorphic facies and behavioral abnormalities (IDDFBA) is a recently described autosomal dominant entity caused by pathogenic variants, mostly de novo, in the FBXO11 gene. It presents in the first years of life with highly variable clinical manifestations. The main features of IDDFBA include borderline-to-severe intellectual disability, behavioral problems, hypotonia, facial dysmorphisms, minor skeletal abnormalities, and recurrent infections. Although eye problems, such as refractive errors, eye misalignment and minor visual changes, have been described in about 48% of patients, a major ocular defect, namely, bilateral optic nerve hypoplasia, has been reported in the literature only once. We report an 8-year-old boy with a novel de novo pathogenic variant in FBXO11 gene (NM_001190274.1: c.1166dup, p.Cys390Metfs∗3) and a complex ophthalmological phenotype, consisting of right microphthalmia, very shallow anterior chamber, and persistent pupillary membrane, right dense nuclear cataract, bilateral optic nerve hypoplasia, and bilateral horizontal manifest nystagmus.

摘要

具有畸形面容和行为异常的智力发育障碍(IDDFBA)是一种最近描述的常染色体显性疾病,由 FBXO11 基因的致病性变异引起,主要为新生突变。它在生命的头几年出现,临床表现高度可变。IDDFBA 的主要特征包括边缘到重度智力残疾、行为问题、肌张力低下、面部畸形、轻微骨骼异常和反复感染。尽管约有 48%的患者出现眼部问题,如屈光不正、眼球斜视和轻微的视觉变化,但文献中仅报道过一次主要眼部缺陷,即双侧视神经发育不良。我们报告了一例 8 岁男孩,他携带 FBXO11 基因的新型新生致病性变异(NM_001190274.1:c.1166dup,p.Cys390Metfs∗3),并伴有复杂的眼部表型,包括右眼小眼球、非常浅的前房和持续的瞳孔膜、右眼致密核性白内障、双侧视神经发育不良和双侧水平显性眼球震颤。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验