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产前外显子组测序鉴定与先天性马蹄内翻足相关的新型 LMX1B 无义变异:一例报告。

Identification of a novel LMX1B nonsense variant associated with congenital talipes equinovarus by prenatal exome sequencing: A case report.

机构信息

Department of Obstetrics and Gynecology, West China Second University Hospital, Sichuan University, Chengdu, Sichuan, China.

Department of Medical Genetics, West China Second University Hospital, Sichuan University, Chengdu, Sichuan, China.

出版信息

Mol Genet Genomic Med. 2024 Jan;12(1):e2303. doi: 10.1002/mgg3.2303. Epub 2023 Nov 6.

Abstract

BACKGROUND

Congenital talipes equinovarus (CTEV) is a rotational foot deformity that affects muscles, bones, connective tissue, and vascular or neurological tissues. The etiology of CTEV is complex and unclear, involving genetic and environmental factors. Nail-patella syndrome is an autosomal dominant disorder caused by variants of the LIM homeobox transcription factor 1 beta gene (LMX1B, OMIM:602575). LMX1B plays a key role in the development of dorsal limb structures, the kidneys, and the eyes, and variants in this gene may manifest as hypoplastic or absent patella, dystrophic nails, and elbow and iliac horn dysplasia; glomerulopathy; and adult-onset glaucoma, respectively. This study aimed to identify pathogenic variants in a fetus with isolated talipes equinovarus diagnosed by ultrasound in the second trimester, whose father exhibited dysplastic nails and congenital absence of bilateral patella.

METHODS

Prenatal whole-exome sequencing (WES) of the fetus and parents was performed to identify the genetic variant responsible for the fetal ultrasound abnormality, followed by validation using Sanger sequencing.

RESULTS

A novel heterozygous nonsense variant in exon 6 of LMX1B (c.844C>T, p.Gln282*) was identified in the fetus and the affected father but was not detected in any unaffected family members. This nonsense variant resulted in a premature termination codon at position 282, which may be responsible for the clinical phenotype through the loss of function of the gene product.

CONCLUSIONS

Our study indicating that a fetus carrying a novel nonsense variant of LMX1B (c.844C>T, p.Gln282*) can exhibit isolated talipes equinovarus, which expands the LMX1B genotypic spectrum and is advantageous for genetic counseling.

摘要

背景

先天性马蹄内翻足(CTEV)是一种影响肌肉、骨骼、结缔组织以及血管或神经组织的旋转足部畸形。CTEV 的病因复杂且尚不明确,涉及遗传和环境因素。指甲髌骨综合征是一种常染色体显性遗传病,由 LIM 同源盒转录因子 1β基因(LMX1B,OMIM:602575)的变异引起。LMX1B 在背侧肢体结构、肾脏和眼睛的发育中起关键作用,该基因的变异可能表现为髌骨发育不全或缺失、指甲营养不良以及肘和髂骨角发育不良;肾小球病;以及成年期开角型青光眼。本研究旨在鉴定在妊娠中期超声诊断为孤立性马蹄内翻足的胎儿中致病变异,其父亲表现为指甲营养不良和双侧髌骨先天性缺失。

方法

对胎儿及其父母进行产前全外显子组测序(WES),以鉴定导致胎儿超声异常的遗传变异,然后使用 Sanger 测序进行验证。

结果

在胎儿和受影响的父亲中发现了 LMX1B 外显子 6 中的一个新的杂合无义变异(c.844C>T,p.Gln282*),但在任何未受影响的家庭成员中均未检测到。该无义变异导致第 282 位的提前终止密码子,可能通过基因产物的功能丧失导致临床表型。

结论

我们的研究表明,携带 LMX1B 新型无义变异(c.844C>T,p.Gln282*)的胎儿可表现为孤立性马蹄内翻足,这扩展了 LMX1B 的基因型谱,有利于遗传咨询。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/eca7/10767578/7b27ac7b7e66/MGG3-12-e2303-g004.jpg

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