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一例 TREX1 基因突变导致的 Aicardi-Goutières 综合征。

A case of Aicardi-Goutières syndrome caused by TREX1 gene mutation.

机构信息

Center for Medical Ultrasound, The Affiliated Suzhou Hospital of Nanjing Medical University, Suzhou Municipal Hospital, Suzhou, China.

Department of Ultrasound, Kunshan Hospital Affiliated to Jiangsu University, Suzhou, China.

出版信息

BMC Pregnancy Childbirth. 2023 Feb 22;23(1):124. doi: 10.1186/s12884-023-05436-5.

Abstract

Aicardi-Goutières syndrome (AGS) is a rare genetic disorder involving the central nervous system and autoimmune abnormalities, leading to severe intellectual and physical disability with poor prognosis. AGS has a phenotype similar to intrauterine viral infection, which often leads to delays in genetic counseling. In this study, we report a case with a prenatal diagnosis of AGS. The first fetal ultrasound detected bilateral lateral ventricle cystic structures, and fetal MRI was performed to identify other signs. The right parietal lobe signal showed cerebral white matter abnormalities, and fetal brain development level was lower than that of normal fetuses of the same gestational age. Whole-exome sequencing revealed that the fetus carried the TREX1:NM_033629.6:exon2:c.294dup:p. C99Mfs*3 variant, suggesting that the c.294dup mutation of the TREX1 gene was the pathogenic mutation site, and the final comprehensive diagnosis was AGS1. In this article, we also reviewed the previous literature for possible phenotypes in the fetus and found that microcephaly and intrauterine growth retardation may be the first and most important markers of the intrauterine phenotype of AGS.

摘要

Aicardi-Goutières 综合征(AGS)是一种罕见的遗传性疾病,涉及中枢神经系统和自身免疫异常,导致严重的智力和身体残疾,预后不良。AGS 的表型类似于宫内病毒感染,这往往导致遗传咨询延迟。本研究报告了一例产前诊断为 AGS 的病例。首次胎儿超声检查发现双侧侧脑室囊性结构,并进行胎儿 MRI 以识别其他征象。右顶叶信号显示脑白质异常,胎儿脑发育水平低于同孕龄正常胎儿。全外显子组测序显示胎儿携带 TREX1:NM_033629.6:exon2:c.294dup:p. C99Mfs*3 变异,提示 TREX1 基因的 c.294dup 突变是致病突变位点,最终综合诊断为 AGS1。本文还回顾了以往文献中可能的胎儿表型,发现小头畸形和宫内生长迟缓可能是 AGS 宫内表型的第一个也是最重要的标志物。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7203/9945611/9f63a894f78a/12884_2023_5436_Fig1_HTML.jpg

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