Expanding MNS1 Heterotaxy Phenotype

被引:0
|
作者
Maraval, Julien [1 ,2 ]
Delahaye-Duriez, Andree [3 ,4 ,5 ]
Racine, Caroline [1 ,2 ]
Bruel, Ange-Line [2 ,6 ]
Denomme-Pichon, Anne-Sophie [2 ,6 ]
Gaudillat, Lea [1 ,2 ]
Thauvin-Robinet, Christel [1 ,2 ,6 ]
Lucain, Marie [2 ,6 ]
Satre, Veronique [7 ,8 ,9 ,10 ]
Coutton, Charles [7 ,8 ,9 ,10 ]
de Sainte Agathe, Jean-Madelaine [11 ]
Keren, Boris [11 ]
Faivre, Laurence [1 ,2 ]
机构
[1] CHU Dijon Bourgogne, Ctr Genet, Ctr Reference Malad Rares Anomalies Dev & Syndrome, FHU TRANSLAD, Dijon, France
[2] Univ Bourgogne Franche Comte, Inserm GAD UMR1231, Dijon, France
[3] Hop Jean Verdier, Hop Univ Paris Seine St Denis, APHP, UF Med Genom & Genet Clin, Bondy, France
[4] Univ Sorbonne Paris Nord, UFR Sante Med Biol Humaine, Bobigny, France
[5] Univ Paris Cite, Inserm UMR1141 NeuroDiderot, Paris, France
[6] CHU Dijon Bourgogne, Un Fonct Innovat Diagnost Genom Malad Rares, Dijon, France
[7] CHU Grenoble, Lab Biol Med Multisites AURAGEN, Grenoble, France
[8] CHU Grenoble Alpes, Lab Genet Chromos, Grenoble, France
[9] Univ Grenoble Alpes, Inst Adv Biosci, Genet Epigenet & Therapies Infertil Team, CNRS UMR2309,Inserm U1209, F-38000 Grenoble, France
[10] GCS AURAGEN, Lyon, France
[11] Sorbonne Univ, Hop Pitie Salpetriere, APHP, Dept Genet Med, Paris, France
关键词
fetus; heterotaxy; MNS1; phenotype; prenatal; recessive; LEFT-RIGHT ASYMMETRY; FUNCTIONAL-CHARACTERIZATION; MUTATIONS; IDENTIFICATION; PREVALENCE; VARIANTS; DEFECTS; GENE;
D O I
10.1002/ajmg.a.63862
中图分类号
Q3 [遗传学];
学科分类号
071007 ; 090102 ;
摘要
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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页数:7
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