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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.

DOI:10.1016/j.jaapos.2022.05.008
PMID:35940561
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。

相似文献

1
New ocular findings in a patient with a novel pathogenic variant in the FBXO11 gene.患者携带 FBXO11 基因新型致病变异的新眼部发现。
J AAPOS. 2022 Oct;26(5):268-270. doi: 10.1016/j.jaapos.2022.05.008. Epub 2022 Aug 5.
2
The first familial case of inherited intellectual developmental disorder with dysmorphic facies and behavioral abnormalities (IDDFBA) with a novel FBXO11 variant.首例携带新型FBXO11变异体的伴有面部畸形和行为异常的遗传性智力发育障碍(IDDFBA)家族病例。
Am J Med Genet A. 2020 Nov;182(11):2788-2792. doi: 10.1002/ajmg.a.61828. Epub 2020 Sep 9.
3
De novo variants in FBXO11 cause a syndromic form of intellectual disability with behavioral problems and dysmorphisms.FBXO11 中的新生变异导致伴有行为问题和发育异常的综合征型智力残疾。
Eur J Hum Genet. 2019 May;27(5):738-746. doi: 10.1038/s41431-018-0292-2. Epub 2019 Jan 24.
4
FBXO11 variants are associated with intellectual disability and variable clinical manifestation in Chinese affected individuals.FBXO11 变异与中国受影响个体的智力残疾和可变临床表现有关。
J Hum Genet. 2024 Aug;69(8):391-400. doi: 10.1038/s10038-024-01255-4. Epub 2024 May 13.
5
De novo FBXO11 mutations are associated with intellectual disability and behavioural anomalies.FBXO11 基因的新生突变与智力障碍和行为异常有关。
Hum Genet. 2018 May;137(5):401-411. doi: 10.1007/s00439-018-1892-1. Epub 2018 May 23.
6
De Novo Variants in the F-Box Protein FBXO11 in 20 Individuals with a Variable Neurodevelopmental Disorder.20 名具有可变神经发育障碍个体中的 F-Box 蛋白 FBXO11 中的从头变异。
Am J Hum Genet. 2018 Aug 2;103(2):305-316. doi: 10.1016/j.ajhg.2018.07.003. Epub 2018 Jul 26.
7
How many phenotypes for the FBXO11 related disease? Report on a new patient with a tricho-rhino-phalangeal like phenotype.FBXO11 相关疾病有多少种表型?报告一例具有毛发-鼻-指(趾)样表型的新患者。
Eur J Med Genet. 2024 Jun;69:104944. doi: 10.1016/j.ejmg.2024.104944. Epub 2024 Apr 26.
8
De novo missense variants in FBXO11 alter its protein expression and subcellular localization.FBXO11 中的从头错义变异改变了其蛋白质表达和亚细胞定位。
Hum Mol Genet. 2022 Feb 3;31(3):440-454. doi: 10.1093/hmg/ddab265.
9
Novel variants likely disrupt DNA binding: molecular modeling in two cases, review of published cases, genotype-phenotype correlation, and phenotypic expansion of the Bosch-Boonstra-Schaaf optic atrophy syndrome.新型变异可能破坏DNA结合:两个案例的分子建模、已发表案例综述、基因型-表型相关性以及博世-布恩斯特拉-沙夫视神经萎缩综合征的表型扩展
Cold Spring Harb Mol Case Stud. 2017 Nov 21;3(6). doi: 10.1101/mcs.a002162. Print 2017 Nov.
10
De novo and inherited TCF20 pathogenic variants are associated with intellectual disability, dysmorphic features, hypotonia, and neurological impairments with similarities to Smith-Magenis syndrome.新生和遗传的 TCF20 致病性变异与智力障碍、发育异常、肌张力减退和神经损伤有关,这些表现与 Smith-Magenis 综合征相似。
Genome Med. 2019 Feb 28;11(1):12. doi: 10.1186/s13073-019-0623-0.

引用本文的文献

1
Proteasomal activation ameliorates neuronal phenotypes linked to FBXO11-deficiency.蛋白酶体激活改善了与FBXO11缺乏相关的神经元表型。
HGG Adv. 2025 Apr 10;6(2):100425. doi: 10.1016/j.xhgg.2025.100425. Epub 2025 Mar 20.
2
FBXO11 variants are associated with intellectual disability and variable clinical manifestation in Chinese affected individuals.FBXO11 变异与中国受影响个体的智力残疾和可变临床表现有关。
J Hum Genet. 2024 Aug;69(8):391-400. doi: 10.1038/s10038-024-01255-4. Epub 2024 May 13.