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患者因发育迟缓、肌张力减退和癫痫就诊,存在染色体易位 t(3;4)(q27;q31.2),伴有 3q27 缺失和 FBXW7 表达减少。

Reciprocal chromosome translocation t(3;4)(q27;q31.2) with deletion of 3q27 and reduced FBXW7 expression in a patient with developmental delay, hypotonia, and seizures.

机构信息

Department of Pediatrics and Child Health, Nihon University School of Medicine, Tokyo, Japan.

Division of Gene Medicine, Graduate School of Medical Science, Tokyo Women's Medical University, Tokyo, Japan.

出版信息

J Hum Genet. 2024 Dec;69(12):639-644. doi: 10.1038/s10038-024-01286-x. Epub 2024 Aug 9.

Abstract

Reciprocal chromosomal translocation is one of genomic variations. When cytogenetically de novo reciprocal translocations are identified in patients with some clinical manifestations, the genes in the breakpoints are considered to be related to the clinical features. In this study, we encountered a patient with severe developmental delay, intractable epilepsy, growth failure, distinctive features, and skeletal manifestations. Conventional karyotyping revealed a de novo translocation described as 46,XY,t(3;4)(q27;q31.2). Chromosomal microarray testing detected a 1.25-Mb microdeletion at 3q27.3q28. Although the skeletal manifestations may have been affected by this deletion, the neurological features of this patient were severe and could not be fully explained by this deletion. Since no genomic copy number aberration was detected on chromosome 4, long-read whole-genome sequencing analysis was performed and a precise breakpoint was confirmed. A 460-bp deletion was detected between the two breakpoints; however, no gene was disrupted. FBXW7, the gene responsible for developmental delay, hypotonia, and impaired language, is in the 0.5-Mb telomeric region. Most of the patient's clinical features were considered consistent with symptoms of FBXW7-related disorders, but were more severe. FBXW7 expression in the immortalized lymphoblasts of the patient was reduced compared to that in controls. Based on these findings, we suspect that FBXW7 is affected by downstream position effects of chromosomal translocations. The severe neurological features of the patient may have been affected not only by the 3q27-q28 deletion but also by impaired expression of FBXW7 derived from the breakage of chromosome 4.

摘要

相互易位是基因组变异之一。当在具有某些临床表现的患者中发现新发生的细胞遗传学相互易位时,认为断点处的基因与临床特征有关。在这项研究中,我们遇到了一位患有严重发育迟缓、难治性癫痫、生长发育不良、特征明显和骨骼表现的患者。常规核型分析显示新发易位描述为 46,XY,t(3;4)(q27;q31.2)。染色体微阵列检测发现 3q27.3q28 处有 1.25Mb 的微缺失。虽然骨骼表现可能受到该缺失的影响,但该患者的神经特征严重,无法完全用该缺失来解释。由于在 4 号染色体上未检测到基因组拷贝数异常,因此进行了长读长全基因组测序分析,并确认了精确的断点。在两个断点之间检测到 460bp 的缺失;然而,没有基因被破坏。FBXW7 是导致发育迟缓、低张力和语言障碍的基因,位于 0.5Mb 端粒区域。患者的大多数临床特征被认为与 FBXW7 相关疾病的症状一致,但更为严重。与对照相比,患者的永生淋巴母细胞中的 FBXW7 表达减少。基于这些发现,我们怀疑 FBXW7 受到染色体易位下游位置效应的影响。患者严重的神经特征不仅受到 3q27-q28 缺失的影响,还受到源自 4 号染色体断裂的 FBXW7 表达受损的影响。

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