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MOGS 复合杂合变异导致先天性糖基化障碍(CDG)IIb。

Compound heterozygous variants in MOGS inducing congenital disorders of glycosylation (CDG) IIb.

机构信息

Pediatric Intensive Care Unit, Anhui Provincial Children's Hospital, 230029, Hefei, P.R. China.

Beijing Chigene Translational Medicine Research Center Co., Ltd, 100875, Beijing, P.R. China.

出版信息

J Hum Genet. 2019 Mar;64(3):265-268. doi: 10.1038/s10038-018-0552-6. Epub 2018 Dec 26.

Abstract

This study is to present two Chinese siblings who were diagnosed with congenital disorders of glycosylation (CDG) IIb because of mannosyl-oligosaccharide glucosidase (MOGS) deficiency. The siblings visited our hospital due to "pulmonary infection". Facial dysmorphism including long eyelashes, blepharophimosis, depressed nasal bridge, and high palate was noted. Head MRI of the elder sister showed increased signals on T1W1, bilateral frontal gyrus stenosis, and thin corpus callosum. Both cases presented progressive hepatomegaly and elevated hepatic enzymes. Low immunoglobulin was discovered in the siblings. Compound heterozygous variants of NM_006302:c.1239_1267dup,p.Asp414Leufs*17, c.544 G > A,p.Gly182Arg, and c.1698C > A,p.Asp566Glu in MOGS were identified. Structural modeling demonstrated that the mutations were pathogenic to MOGS. Our study enriched the genetic and phenotypic spectrum of MOGS-CDG, and for children with facial dysmorphism, postnatal dyspnea, seizures, motor developmental delay, hypotonia, and immunological or gastrointestinal dysfunction, this disease should be highly suspected.

摘要

本研究介绍了两名因甘露糖基-寡糖葡萄糖苷酶(MOGS)缺乏而被诊断为先天性糖基化障碍(CDG)IIb 的中国同胞。这对同胞因“肺部感染”来我院就诊。姐姐存在睫毛长、睑裂狭小、鼻梁凹陷和高腭弓等面部畸形。姐姐的头部 MRI 显示 T1W1 信号增高,双侧额回狭窄,胼胝体变薄。两例均表现为进行性肝肿大和肝酶升高。同胞均发现免疫球蛋白降低。在 MOGS 中发现了 NM_006302:c.1239_1267dup,p.Asp414Leufs*17、c.544 G > A,p.Gly182Arg 和 c.1698C > A,p.Asp566Glu 的复合杂合变异。结构建模表明这些突变对 MOGS 具有致病性。本研究丰富了 MOGS-CDG 的遗传和表型谱,对于有面部畸形、出生后呼吸困难、癫痫、运动发育迟缓、肌张力低下以及免疫或胃肠道功能障碍的儿童,应高度怀疑该病。

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