LAMB1双等位变异导致无水脑畸形:一种罕见的畸形脑病的严重表型。

IF 0.8 Q4 PEDIATRICS
AJP Reports Pub Date : 2021-01-01 Epub Date: 2021-02-01 DOI:10.1055/s-0040-1722728
Kuntal Sen, Shagun Kaur, David W Stockton, Mary Nyhuis, Jacquelyn Roberson
{"title":"LAMB1双等位变异导致无水脑畸形:一种罕见的畸形脑病的严重表型。","authors":"Kuntal Sen,&nbsp;Shagun Kaur,&nbsp;David W Stockton,&nbsp;Mary Nyhuis,&nbsp;Jacquelyn Roberson","doi":"10.1055/s-0040-1722728","DOIUrl":null,"url":null,"abstract":"<p><p><b>Case Report</b>  A 32-year-old female with a history of three prior pregnancy losses presented for genetic testing following an ultrasonography diagnosis of fetal hydranencephaly. Baby was born via C-section and was noted to have a head circumference of 48 cm, in addition to ocular and cardiac anomalies and dysmorphic features. Whole genome sequencing revealed a homozygous variant in <i>LAMB1</i> gene. <b>Discussion</b>  The pathobiogenesis of hydranencephaly is incompletely understood and is attributed to vascular, infectious, or genetic etiology. Herein we present <i>LAMB1</i> as a monogenic cause of fetal hydranencephaly which was incompatible with life. Previously, <i>LAMB1</i> -associated phenotype consisted of cobblestone lissencephaly and hydrocephalus, developmental delay, and seizures. Our proband expands the phenotypic spectrum of this malformative encephalopathy.</p>","PeriodicalId":7645,"journal":{"name":"AJP Reports","volume":"11 1","pages":"e26-e28"},"PeriodicalIF":0.8000,"publicationDate":"2021-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1055/s-0040-1722728","citationCount":"4","resultStr":"{\"title\":\"Biallelic Variants in <i>LAMB1</i> Causing Hydranencephaly: A Severe Phenotype of a Rare Malformative Encephalopathy.\",\"authors\":\"Kuntal Sen,&nbsp;Shagun Kaur,&nbsp;David W Stockton,&nbsp;Mary Nyhuis,&nbsp;Jacquelyn Roberson\",\"doi\":\"10.1055/s-0040-1722728\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p><b>Case Report</b>  A 32-year-old female with a history of three prior pregnancy losses presented for genetic testing following an ultrasonography diagnosis of fetal hydranencephaly. Baby was born via C-section and was noted to have a head circumference of 48 cm, in addition to ocular and cardiac anomalies and dysmorphic features. Whole genome sequencing revealed a homozygous variant in <i>LAMB1</i> gene. <b>Discussion</b>  The pathobiogenesis of hydranencephaly is incompletely understood and is attributed to vascular, infectious, or genetic etiology. Herein we present <i>LAMB1</i> as a monogenic cause of fetal hydranencephaly which was incompatible with life. Previously, <i>LAMB1</i> -associated phenotype consisted of cobblestone lissencephaly and hydrocephalus, developmental delay, and seizures. Our proband expands the phenotypic spectrum of this malformative encephalopathy.</p>\",\"PeriodicalId\":7645,\"journal\":{\"name\":\"AJP Reports\",\"volume\":\"11 1\",\"pages\":\"e26-e28\"},\"PeriodicalIF\":0.8000,\"publicationDate\":\"2021-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.1055/s-0040-1722728\",\"citationCount\":\"4\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"AJP Reports\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1055/s-0040-1722728\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2021/2/1 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q4\",\"JCRName\":\"PEDIATRICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"AJP Reports","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1055/s-0040-1722728","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2021/2/1 0:00:00","PubModel":"Epub","JCR":"Q4","JCRName":"PEDIATRICS","Score":null,"Total":0}
引用次数: 4

摘要

病例报告一名32岁女性,有三次流产史,超声诊断胎儿无脑畸形后进行基因检测。婴儿通过剖腹产出生,并注意到头围为48厘米,此外还有眼和心脏异常和畸形特征。全基因组测序显示LAMB1基因的纯合子变异。无脑畸形的发病机制尚不完全清楚,可归因于血管、感染或遗传病因。在这里,我们提出LAMB1作为胎儿无脑畸形的单基因原因,与生命不相容。以前,LAMB1相关表型包括卵石状无脑畸形和脑积水,发育迟缓和癫痫发作。我们的先证者扩展了这种畸形脑病的表型谱。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Biallelic Variants in <i>LAMB1</i> Causing Hydranencephaly: A Severe Phenotype of a Rare Malformative Encephalopathy.

Biallelic Variants in LAMB1 Causing Hydranencephaly: A Severe Phenotype of a Rare Malformative Encephalopathy.

Case Report  A 32-year-old female with a history of three prior pregnancy losses presented for genetic testing following an ultrasonography diagnosis of fetal hydranencephaly. Baby was born via C-section and was noted to have a head circumference of 48 cm, in addition to ocular and cardiac anomalies and dysmorphic features. Whole genome sequencing revealed a homozygous variant in LAMB1 gene. Discussion  The pathobiogenesis of hydranencephaly is incompletely understood and is attributed to vascular, infectious, or genetic etiology. Herein we present LAMB1 as a monogenic cause of fetal hydranencephaly which was incompatible with life. Previously, LAMB1 -associated phenotype consisted of cobblestone lissencephaly and hydrocephalus, developmental delay, and seizures. Our proband expands the phenotypic spectrum of this malformative encephalopathy.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
AJP Reports
AJP Reports PEDIATRICS-
CiteScore
2.20
自引率
0.00%
发文量
30
审稿时长
12 weeks
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信