Prenatal Diagnosis of Jeune Syndrome Caused by Compound Heterozygous Variants in

DYNC2H1 Jeune syndrome asphyxiating thoracic dystrophy lethal skeletal dysplasia life-limiting skeletal dysplasia prenatal diagnosis

Journal

Genes
ISSN: 2073-4425
Titre abrégé: Genes (Basel)
Pays: Switzerland
ID NLM: 101551097

Informations de publication

Date de publication:
27 07 2022
Historique:
received: 11 07 2022
revised: 24 07 2022
accepted: 26 07 2022
entrez: 27 7 2022
pubmed: 28 7 2022
medline: 29 7 2022
Statut: epublish

Résumé

Skeletal dysplasias (SDs) are a large, heterogeneous group of mostly genetic disorders that affect the bones and cartilage, resulting in abnormal growth and development of skeletal structures. The high clinical and genetic diversity in SDs cause difficulties in prenatal diagnosis. To establish a correct prognosis and better management, it is very important to distinguish SDs with poor life-limiting prognosis or lethal SDs from other ones. Bad prognosis in foetuses is assessed on the basis of the size of the thorax, lung volumes, long bones’ length, bones’ echogenicity, bones’ angulation or presented fractures, and the concomitant presence of non-immune hydrops or visceral abnormalities. To confirm SD diagnosis and perform family genetic consultation, rapid molecular diagnostics are needed; therefore, the NGS method using a panel of genes corresponding to SD or whole-exome sequencing (WES) is commonly used. We report a case of a foetus showing long bones’ shortening and a narrow chest with short ribs, diagnosed prenatally with asphyxiating thoracic dystrophy, also known as Jeune syndrome (ATD; OMIM 208500), caused by compound heterozygous variants in the DYNC2H1 gene, identified by prenatally performed rapid-WES analysis. The missense variants in the DYNC2H1 gene were inherited from the mother (c.7289T>C; p.Ile2430Thr) and from the father (c.12716T>G; p.Leu4239Arg). The DYNC2H1 gene is one of at least 17 ATD-associated genes. This disorder belongs to the ninth group of SD, ciliopathies with major skeletal involvement. An extremely narrow, bell-shaped chest, and abnormalities of the kidneys, liver, and retinas were observed in most cases of ATD. Next to lethal and severe forms, clinically mild forms have also been reported. A diagnosis of ATD is important to establish the prognosis and management for the patient, as well as the recurrence risk for the family.

Identifiants

pubmed: 35893076
pii: genes13081339
doi: 10.3390/genes13081339
pmc: PMC9332837
pii:
doi:

Substances chimiques

DYNC2H1 protein, human 0
Cytoplasmic Dyneins EC 3.6.4.2

Types de publication

Case Reports Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

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Auteurs

Agnieszka Stembalska (A)

Department of Genetics, Medical University, Marcinkowskiego 1, 50-368 Wroclaw, Poland.

Małgorzata Rydzanicz (M)

Department of Medical Genetics, Medical University of Warsaw, Adolfa Pawińskiego 3c, 02-106 Warsaw, Poland.

Magdalena Klaniewska (M)

Department of Family and Pediatric Nursing, Medical University, Bartla 5, 51-618 Wroclaw, Poland.

Lech Dudarewicz (L)

Department of Genetics, Polish Mother's Memorial Hospital-Research Institute, Rzgowska 281/289, 93-338 Lodz, Poland.

Agnieszka Pollak (A)

Department of Medical Genetics, Medical University of Warsaw, Adolfa Pawińskiego 3c, 02-106 Warsaw, Poland.

Mateusz Biela (M)

Department of Family and Pediatric Nursing, Medical University, Bartla 5, 51-618 Wroclaw, Poland.

Piotr Stawinski (P)

Department of Medical Genetics, Medical University of Warsaw, Adolfa Pawińskiego 3c, 02-106 Warsaw, Poland.

Rafal Ploski (R)

Department of Medical Genetics, Medical University of Warsaw, Adolfa Pawińskiego 3c, 02-106 Warsaw, Poland.

Robert Smigiel (R)

Department of Family and Pediatric Nursing, Medical University, Bartla 5, 51-618 Wroclaw, Poland.

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