Association of Meier-Gorlin and microcephalic osteodysplastic primordial dwarfism type II clinical features in an individual with CDK5RAP2 primary microcephaly.


Journal

European journal of medical genetics
ISSN: 1878-0849
Titre abrégé: Eur J Med Genet
Pays: Netherlands
ID NLM: 101247089

Informations de publication

Date de publication:
May 2023
Historique:
received: 24 11 2022
revised: 27 01 2023
accepted: 22 02 2023
medline: 3 4 2023
pubmed: 27 2 2023
entrez: 26 2 2023
Statut: ppublish

Résumé

Autosomal recessive primary microcephaly type 3 (MCPH3) caused by pathogenic variations in CDK5RAP2, is characterized by sensorineural hearing loss, abnormality of skin pigmentation, ocular defects and severe microcephaly associated with neurodevelopmental delay. In this study, we expand the phenotype of MCPH3 as we describe a 10-year-old girl with a biallelic exonic frameshift variant in CDK5RAP2 displaying previously unreported features usually associated with Meier-Gorlin and microcephalic osteodysplastic primordial dwarfism type II (MOPDII). We further describe the clinical phenotype of this form of centrosomal-based primary microcephaly and emphasize the importance of skeletal defect screening in affected individuals.

Identifiants

pubmed: 36842471
pii: S1769-7212(23)00039-3
doi: 10.1016/j.ejmg.2023.104733
pii:
doi:

Substances chimiques

CDK5RAP2 protein, human 0
Nerve Tissue Proteins 0
Cell Cycle Proteins 0

Types de publication

Case Reports Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

104733

Informations de copyright

Copyright © 2023 Elsevier Masson SAS. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of competing interest The authors declare no competing interests.

Auteurs

Quentin Sabbagh (Q)

Montpellier University, Service de Génétique Clinique, Centre de Compétence Maladies Osseuses Constitutionnelles, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Mylène Tharreau (M)

Montpellier University, Department of Molecular Genetics and Cytogenomics, Rare and Autoinflammatory Diseases Unit, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Camille Cenni (C)

Montpellier University, Department of Molecular Genetics and Cytogenomics, Rare and Autoinflammatory Diseases Unit, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Elodie Sanchez (E)

Montpellier University, Department of Molecular Genetics and Cytogenomics, Rare and Autoinflammatory Diseases Unit, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Nathalie Ruiz-Pallares (N)

Montpellier University, Department of Molecular Genetics and Cytogenomics, Rare and Autoinflammatory Diseases Unit, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Fanny Alkar (F)

Montpellier University, Service de Génétique Clinique, Centre de Compétence Maladies Osseuses Constitutionnelles, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Cyril Amouroux (C)

Montpellier University, Pediatric Endocrinology and Nephrology Unit, Rare Diseases Competence Center for Bone Mineral Disease and Skeletal Dysplasia, OSCAR Network, Montpellier University Hospital, Montpellier, France.

Stéphanie David (S)

Montpellier University, Département d'Imagerie Pédiatrique, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Olivier Prodhomme (O)

Montpellier University, Département d'Imagerie Pédiatrique, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Nicolas Leboucq (N)

Montpellier University, Département d'Imagerie Pédiatrique, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Isabelle Meunier (I)

Montpellier University, Département dOphtalmologie, Centre de Référence Maladies Sensorielles Génétiques, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Didier Bessis (D)

Montpellier University, Département de Dermatologie, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Alexandre Theron (A)

Montpellier University, Service d'Onco-hématologie Pédiatrique, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Mouna Barat-Houari (M)

Montpellier University, Department of Molecular Genetics and Cytogenomics, Rare and Autoinflammatory Diseases Unit, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.

Marjolaine Willems (M)

Montpellier University, Service de Génétique Clinique, Centre de Compétence Maladies Osseuses Constitutionnelles, Centre Hospitalier Universitaire de Montpellier, Montpellier, France; Montpellier University, Institute for Neurosciences of Montpellier, INSERM, Montpellier, France. Electronic address: m-willems@chu-montpellier.fr.

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Classifications MeSH