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枕部脑膨出、多小脑回、眼部异常和蚓部发育异常:诺布洛赫综合征的产前标志物

Occipital Cephalocele, Polymicrogyria, Ocular Anomaly and Vermian Dysplasia: Prenatal Markers for Knobloch Syndrome.

作者信息

Carmant Laurence Sophie, Miller Elka, Chitayat David, Hedges Stephanie, McGivern Bobbi, Harris Kimberly, Chong Karen, Shinar Shiri

机构信息

Division of Maternal Fetal Medicine, Department of Obstetrics and Gynaecology, Mount Sinai Hospital, University of Toronto, Toronto, Canada.

Department of Diagnostic and Interventional Radiology, The Hospital for Sick Children, University of Toronto, Toronto, Canada.

出版信息

Prenat Diagn. 2025 Jul;45(8):1074-1077. doi: 10.1002/pd.6838. Epub 2025 Jun 16.

Abstract

Knobloch Syndrome-1 is a rare autosomal recessive disorder typically diagnosed postnatally and characterized by occipital encephalocele, high myopia, and vitreoretinal degeneration. We describe a fetus with a constellation of prenatal neuroimaging findings, including occipital cephalocele, vermian dysplasia, bilateral polymicrogyria, and ocular elongation, that prompted genetic investigation. Trio exome sequencing identified biallelic pathogenic variants in COL18A1, confirming the diagnosis of Knobloch Syndrome-1. This case highlights how advanced fetal neuroimaging and prenatal exome sequencing can facilitate early recognition of syndromes like Knobloch, and underscores the importance of considering COL18A1-related disorders when multiple central nervous system anomalies are detected prenatally.

摘要

诺布罗赫综合征1型是一种罕见的常染色体隐性疾病,通常在出生后诊断,其特征为枕部脑膨出、高度近视和玻璃体视网膜变性。我们描述了一名胎儿,其具有一系列产前神经影像学检查结果,包括枕部脑膨出、蚓部发育异常、双侧多小脑回畸形和眼轴延长,这些结果促使进行基因检测。三联体外显子组测序在COL18A1基因中鉴定出双等位基因致病性变异,确诊为诺布罗赫综合征1型。该病例突出了先进的胎儿神经影像学检查和产前外显子组测序如何有助于早期识别诺布罗赫综合征等疾病,并强调了在产前检测到多个中枢神经系统异常时考虑COL18A1相关疾病的重要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/57ee/12254427/c2d873cef99d/PD-45-1074-g001.jpg

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