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在一个伊朗的近亲家庭中鉴定出 FGF3 基因的纯合移码突变:伊朗首例迷路发育不全、小耳畸形和小牙畸形综合征的报告和文献复习。

Identification of a homozygous frameshift mutation in the FGF3 gene in a consanguineous Iranian family: First report of labyrinthine aplasia, microtia, and microdontia syndrome in Iran and literature review.

机构信息

Genetics Research Center, University of Social Welfare and Rehabilitation Sciences, Koodakyar Alley, Daneshjoo Blvd., Evin St., 1985713834, Tehran, Iran.

出版信息

Mol Genet Genomic Med. 2023 May;11(5):e2168. doi: 10.1002/mgg3.2168. Epub 2023 Mar 19.

Abstract

BACKGROUND

To date, over 400 syndromes with hearing impairment have been identified which altogether constitute almost 30% of hereditary hearing loss (HL) cases around the globe. Manifested as complete or partial labyrinthine aplasia (severe malformations of the inner ear structure), type I microtia (smaller outer ear with shortened auricles), and microdontia (small and widely spaced teeth), labyrinthine aplasia, microtia, and microdontia (LAMM) syndrome (OMIM 610706) is an extremely rare autosomal recessive condition caused by bi-allelic mutations in the FGF3 gene.

METHODS

Using the whole-exome sequencing (WES) data of the proband, we analyzed a consanguineous Iranian family with three affected members presenting with congenital bilateral HL, type I microtia, and microdontia.

RESULTS

We discovered the homozygous deletion c.45delC in the first exon of the FGF3 gene, overlapping a 38.72 Mb homozygosity region in chromosome 11. Further investigations using Sanger sequencing revealed that this variant co-segregated with the phenotype observed in the family.

CONCLUSION

Here, we report the first identified case of LAMM syndrome in Iran, and by identifying a frameshift variant in the first exon of the FGF3 gene, our result will help better clarify the phenotype-genotype relation of LAMM syndrome.

摘要

背景

迄今为止,已经发现了超过 400 种伴有听力障碍的综合征,这些综合征加起来构成了全球近 30%的遗传性听力损失(HL)病例。内耳结构完全或部分发育不全(严重畸形)、I 型小耳畸形(外耳较小,耳轮短缩)和小牙症(牙齿小而稀疏)是内耳发育不全、小耳畸形和小牙症(LAMM)综合征(OMIM 610706)的主要表现,这种疾病极其罕见,是由 FGF3 基因的双等位基因突变引起的常染色体隐性遗传病。

方法

利用先证者的外显子组测序(WES)数据,我们对一个有血缘关系的伊朗家庭进行了分析,该家庭的 3 名成员均患有先天性双侧 HL、I 型小耳畸形和小牙症。

结果

我们发现 FGF3 基因第一外显子中的纯合缺失 c.45delC,与染色体 11 上的一个 38.72 Mb 纯合区域重叠。使用 Sanger 测序进行的进一步研究表明,该变体与家族中观察到的表型共分离。

结论

我们在伊朗首次报道了 LAMM 综合征病例,通过鉴定 FGF3 基因第一外显子中的移码变异,我们的研究结果将有助于更好地阐明 LAMM 综合征的表型-基因型关系。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/ba59/10178790/e22bf81409cc/MGG3-11-e2168-g002.jpg

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