Maraval Julien, Delahaye-Duriez Andrée, Racine Caroline, Bruel Ange-Line, Denommé-Pichon Anne-Sophie, Gaudillat Léa, Thauvin-Robinet Christel, Lucain Marie, Satre Véronique, Coutton Charles, de Sainte Agathe Jean-Madelaine, Keren Boris, Faivre Laurence
Centre de Génétique, FHU-TRANSLAD, CHU Dijon Bourgogne, Centre de Référence Maladies Rares Anomalies du Développement et Syndromes Malformatifs, Dijon, France.
Inserm UMR1231 GAD, Université Bourgogne-Franche Comté, Dijon, France.
Am J Med Genet A. 2025 Jan;197(1):e63862. doi: 10.1002/ajmg.a.63862. Epub 2024 Sep 5.
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.
MNS1(减数分裂特异性核结构蛋白1基因)编码一种与运动性纤毛功能和精子鞭毛组装有关的结构蛋白。迄今为止,两种不同的纯合MNS1变异体与常染色体隐性内脏异位(MIM#618948)有关。一名法国个体被鉴定出MNS1基因存在复合杂合变异体。通过基因匹配器发起了一项合作呼吁,以描述患有这种罕见综合征的新病例,从而发现了另一个家系。首例患者为一名女性,表现为完全性内脏转位及异常症状,包括严重近视和10颗恒牙先天性缺失。发现她携带MNS1基因的复合杂合移码和无义变异体。第二例和第三例患者是同胞胎儿,分别携带MNS1基因的纯合框内缺失变异体和GLDN基因的纯合错义变异体。尸检显示为一种复杂的产前畸形综合征。我们在此补充了患有极罕见的MNS1相关疾病的新病例,并对所有已发表的病例进行了综述。