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一名患有常染色体隐性智力发育障碍和自闭症女孩的基因诊断与表型分析

Genetic Diagnostics and Phenotypic Profiling of a Girl With Autosomal Recessive Intellectual Developmental Disorder and Autism.

作者信息

Lakatosova Silvia, Repiska Gabriela, Valachova Alica, Raskova Barbara, Belica Ivan, Patrovic Lukas, Ostatnikova Daniela, Konecny Michal

机构信息

Genetics, Institute of Physiology, Academic Center for Autism Research, Faculty of Medicine, Comenius University in Bratislava, Bratislava, SVK.

Medical Genetics, Faculty Hospital Trenčín, Trenčín, SVK.

出版信息

Cureus. 2024 Nov 25;16(11):e74379. doi: 10.7759/cureus.74379. eCollection 2024 Nov.

DOI:10.7759/cureus.74379
PMID:39723281
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11669039/
Abstract

In this article, we present a case study of a five-year-old girl with autism and developmental delay, conducted at the Academic Center for Autism Research in Bratislava, Slovakia. The girl was diagnosed using Autism Diagnostic Observation Schedule-Second Edition (ADOS-2) and Autism Diagnostic Interview-Revised (ADI-R) instruments and met the criteria for autism spectrum disorder. Intellectual functioning was in the markedly below-average range, as indicated by the Snijders-Oomen Nonverbal Intelligence Test-Revised (SON-R) examination, and her level of adaptive functioning was significantly reduced. Neurological signs included atypical leukoencephalopathy, hypotonia, sensorineural hearing loss, and sleep disturbances. The patient underwent genetic testing, including microarray-based copy number variation (CNV) detection, which yielded negative results. However, whole exome sequencing (WES) analysis pointed out a damaging homozygous variant in the EIF3F (Eukaryotic Translation Initiation Factor 3 Subunit F) gene, confirming the diagnosis of intellectual developmental disorder autosomal recessive 67. Segregation analysis in the family revealed that the asymptomatic parents were carriers of the pathogenic variant in EIF3F. Our study contributes to the phenotypic profiling of this rare syndromic neurodevelopmental disorder and points out the irreplaceability of WES analysis in genetic diagnostics of autism and developmental delay. This appeals to the need for WES to be accepted as a routine diagnostic tool in Slovakia.

摘要

在本文中,我们展示了一个对患有自闭症和发育迟缓的五岁女孩进行的案例研究,该研究在斯洛伐克布拉迪斯拉发的自闭症研究学术中心开展。该女孩通过使用《自闭症诊断观察量表第二版》(ADOS-2)和《自闭症诊断访谈修订版》(ADI-R)工具进行诊断,符合自闭症谱系障碍的标准。如修订后的斯尼德斯-奥门非言语智力测验(SON-R)所示,其智力功能处于明显低于平均水平的范围,且她的适应性功能水平显著降低。神经学体征包括非典型性白质脑病、肌张力减退、感音神经性听力损失和睡眠障碍。患者接受了基因检测,包括基于微阵列的拷贝数变异(CNV)检测,结果为阴性。然而,全外显子组测序(WES)分析指出真核翻译起始因子3亚基F(EIF3F)基因存在有害的纯合变异,确诊为常染色体隐性遗传性智力发育障碍67型。对该家庭的分离分析显示,无症状的父母是EIF3F致病变异的携带者。我们的研究有助于对这种罕见的综合征性神经发育障碍进行表型分析,并指出WES分析在自闭症和发育迟缓的基因诊断中具有不可替代的作用。这呼吁在斯洛伐克将WES作为一种常规诊断工具予以接受。

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本文引用的文献

1
EIF3F-related neurodevelopmental disorder: refining the phenotypic and expanding the molecular spectrum.EIF3F 相关神经发育障碍:细化表型并扩大分子谱。
Orphanet J Rare Dis. 2021 Mar 18;16(1):136. doi: 10.1186/s13023-021-01744-1.
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Gene discoveries in autism are biased towards comorbidity with intellectual disability.自闭症相关基因的发现偏向于与智力障碍共病。
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Quantifying the contribution of recessive coding variation to developmental disorders.
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SCA8 RAN polySer protein preferentially accumulates in white matter regions and is regulated by eIF3F.SCA8 型 RAN 聚合酶丝氨酸蛋白优先在白质区域积累,并受 eIF3F 调控。
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Unique transcriptome patterns of the white and grey matter corroborate structural and functional heterogeneity in the human frontal lobe.白质和灰质独特的转录组模式证实了人类额叶的结构和功能异质性。
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eIF3f: a central regulator of the antagonism atrophy/hypertrophy in skeletal muscle.eIF3f:骨骼肌萎缩/肥大拮抗作用的核心调节因子。
Int J Biochem Cell Biol. 2013 Oct;45(10):2158-62. doi: 10.1016/j.biocel.2013.06.001. Epub 2013 Jun 13.
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Decreased expression of eukaryotic initiation factor 3f deregulates translation and apoptosis in tumor cells.真核生物起始因子3f的表达降低会导致肿瘤细胞中的翻译和凋亡失调。
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