扩大 MNS1 异位表型。

IF 1.7 4区 生物学 Q3 GENETICS & HEREDITY
Julien Maraval, Andrée Delahaye-Duriez, Caroline Racine, Ange-Line Bruel, Anne-Sophie Denommé-Pichon, Léa Gaudillat, Christel Thauvin-Robinet, Marie Lucain, Véronique Satre, Charles Coutton, Jean-Madelaine de Sainte Agathe, Boris Keren, Laurence Faivre
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引用次数: 0

摘要

MNS1(减数分裂特异性核结构蛋白-1 基因)编码一种与运动纤毛功能和精子鞭毛组装有关的结构蛋白。迄今为止,有两种不同的 MNS1 同源变异与常染色体隐性内脏异位症(MIM#618948)有关。一名法国人被鉴定为 MNS1 基因复合杂合变异体。通过 GeneMatcher 提出了一项合作呼吁,以描述这种罕见综合征的新病例,从而确定了另一个家庭。第一例患者是一名女性,表现为完全性坐位倒置和异常症状,包括严重近视和 10 颗恒牙缺失。她被发现携带 MNS1 的复合杂合变异框移和无义变异。第二名和第三名患者是同胞胎儿,分别携带 MNS1 的同卵框内缺失变异和 GLDN 的同卵错义变异。尸检结果显示这是一种复杂的产前畸形综合征。我们在此补充了与 MNS1 相关的超罕见疾病的新病例,并对所有已发表的病例进行了回顾。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Expanding MNS1 Heterotaxy Phenotype.

MNS1 (meiosis-specific nuclear structural protein-1 gene) encodes a structural protein implicated in motile ciliary function and sperm flagella assembly. To date, two different homozygous MNS1 variants have been associated with autosomal recessive visceral heterotaxy (MIM#618948). A French individual was identified with compound heterozygous variants in the MNS1 gene. A collaborative call was proposed via GeneMatcher to describe new cases with this rare syndrome, leading to the identification of another family. The first patient was a female presenting complete situs inversus and unusual symptoms, including severe myopia and dental agenesis of 10 permanent teeth. She was found to carry compound heterozygous frameshift and nonsense variants in MNS1. The second and third patients were sibling fetuses with homozygous in-frame deletion variants in MNS1 and homozygous missense variants in GLDN. Autopsies revealed a complex prenatal malformation syndrome. We add here new cases with the ultra-rare MNS1-related disorder and provide a review of all published individuals.

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来源期刊
CiteScore
3.50
自引率
5.00%
发文量
432
审稿时长
2-4 weeks
期刊介绍: The American Journal of Medical Genetics - Part A (AJMG) gives you continuous coverage of all biological and medical aspects of genetic disorders and birth defects, as well as in-depth documentation of phenotype analysis within the current context of genotype/phenotype correlations. In addition to Part A , AJMG also publishes two other parts: Part B: Neuropsychiatric Genetics , covering experimental and clinical investigations of the genetic mechanisms underlying neurologic and psychiatric disorders. Part C: Seminars in Medical Genetics , guest-edited collections of thematic reviews of topical interest to the readership of AJMG .
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