Variant recurrence confirms the existence of a FBXO31-related spastic-dystonic cerebral palsy syndrome.


Journal

Annals of clinical and translational neurology
ISSN: 2328-9503
Titre abrégé: Ann Clin Transl Neurol
Pays: United States
ID NLM: 101623278

Informations de publication

Date de publication:
04 2021
Historique:
revised: 20 02 2021
received: 26 01 2021
accepted: 22 02 2021
pubmed: 7 3 2021
medline: 12 1 2022
entrez: 6 3 2021
Statut: ppublish

Résumé

The role of genetics in the causation of cerebral palsy has become the focus of many studies aiming to unravel the heterogeneous etiology behind this frequent neurodevelopmental disorder. A recent paper reported two unrelated children with a clinical diagnosis of cerebral palsy, who carried the same de novo c.1000G > A (p.Asp334Asn) variant in FBXO31, encoding a widely studied tumor suppressor not previously implicated in monogenic disease. We now identified a third individual with the recurrent FBXO31 de novo missense variant, featuring a spastic-dystonic phenotype. Our data confirm a link between variant FBXO31 and an autosomal dominant neurodevelopmental disorder characterized by prominent motor dysfunction.

Identifiants

pubmed: 33675180
doi: 10.1002/acn3.51335
pmc: PMC8045898
doi:

Substances chimiques

F-Box Proteins 0
FBXO31 protein, human 0
Tumor Suppressor Proteins 0

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

951-955

Subventions

Organisme : NHLBI NIH HHS
ID : R00 HL143036
Pays : United States

Informations de copyright

© 2021 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.

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Auteurs

Ivana Dzinovic (I)

Institute of Neurogenomics, Helmholtz Zentrum München, Munich, Germany.

Matej Škorvánek (M)

Department of Neurology, P.J. Safarik University, Kosice, Slovak Republic.
Department of Neurology, University Hospital of L. Pasteur, Kosice, Slovak Republic.

Petra Pavelekova (P)

Department of Neurology, P.J. Safarik University, Kosice, Slovak Republic.
Department of Neurology, University Hospital of L. Pasteur, Kosice, Slovak Republic.

Chen Zhao (C)

Institute of Neurogenomics, Helmholtz Zentrum München, Munich, Germany.

Boris Keren (B)

Department of Genetics, Pitié-Salpêtrière Hospital, APHP.Sorbonne Université, Paris, France.

Sandra Whalen (S)

UF de Génétique Clinique et Centre de Référence Anomalies du Développement et Syndromes Malformatifs, APHP.Sorbonne Université, Hôpital Armand Trousseau, Paris, France.

Somayeh Bakhtiari (S)

Pediatric Movement Disorders Program, Division of Pediatric Neurology, Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, Arizona, USA.
Departments of Child Health, Neurology, and Cellular & Molecular Medicine, and Program in Genetics, University of Arizona College of Medicine-Phoenix, Phoenix, Arizona, USA.

Sheng Chih Jin (S)

Department of Genetics, Washington University School of Medicine, St Louis, Missouri, USA.

Michael C Kruer (MC)

Pediatric Movement Disorders Program, Division of Pediatric Neurology, Barrow Neurological Institute, Phoenix Children's Hospital, Phoenix, Arizona, USA.
Departments of Child Health, Neurology, and Cellular & Molecular Medicine, and Program in Genetics, University of Arizona College of Medicine-Phoenix, Phoenix, Arizona, USA.

Robert Jech (R)

Department of Neurology, Charles University, 1st Faculty of Medicine, General University Hospital, Prague, Czech Republic.

Juliane Winkelmann (J)

Institute of Neurogenomics, Helmholtz Zentrum München, Munich, Germany.
Institute of Human Genetics, Technical University of Munich, Munich, Germany.
Lehrstuhl für Neurogenetik, Technische Universität München, Munich, Germany.
Munich Cluster for Systems Neurology, SyNergy, Munich, Germany.

Michael Zech (M)

Institute of Neurogenomics, Helmholtz Zentrum München, Munich, Germany.
Institute of Human Genetics, Technical University of Munich, Munich, Germany.

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Classifications MeSH