The clinical presentation caused by truncating CHD8 variants.


Journal

Clinical genetics
ISSN: 1399-0004
Titre abrégé: Clin Genet
Pays: Denmark
ID NLM: 0253664

Informations de publication

Date de publication:
07 2019
Historique:
received: 21 02 2019
revised: 29 03 2019
accepted: 10 04 2019
pubmed: 20 4 2019
medline: 25 7 2020
entrez: 20 4 2019
Statut: ppublish

Résumé

Variants in the chromodomain helicase DNA-binding protein 8 (CHD8) have been associated with intellectual disability (ID), autism spectrum disorders (ASDs) and overgrowth and CHD8 is one of the causative genes for OGID (overgrowth and ID). We investigated 25 individuals with CHD8 protein truncating variants (PTVs), including 10 previously unreported patients and found a male to female ratio of 2.7:1 (19:7) and a pattern of common features: macrocephaly (62.5%), tall stature (47%), developmental delay and/or intellectual disability (81%), ASDs (84%), sleep difficulties (50%), gastrointestinal problems (40%), and distinct facial features. Most of the individuals in this cohort had moderate-to-severe ID, some had regression of speech (37%), seizures (27%) and hypotonia (27%) and two individuals were non-ambulant. Our study shows that haploinsufficiency of CHD8 is associated with a distinctive OGID syndrome with pronounced autistic traits and supports a sex-dependent penetrance of CHD8 PTVs in humans.

Identifiants

pubmed: 31001818
doi: 10.1111/cge.13554
doi:

Substances chimiques

CHD8 protein, human 0
DNA-Binding Proteins 0
Transcription Factors 0

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

72-84

Subventions

Organisme : Medical Research Council
ID : MC_PC_16018
Pays : United Kingdom
Organisme : Department of Health
ID : HICF-1009-003
Pays : United Kingdom
Organisme : Wellcome Trust
ID : WT098051
Pays : United Kingdom

Informations de copyright

© 2019 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.

Auteurs

Sofia Douzgou (S)

Manchester Centre for Genomic Medicine, St Mary's Hospital, Manchester University Hospitals NHS Foundation Trust, Manchester Academic Health Sciences Centre, Manchester, UK.
Division of Evolution and Genomic Sciences, School of Biological Sciences, University of Manchester, Manchester, UK.

Hui Wen Liang (HW)

Division of Evolution and Genomic Sciences, School of Biological Sciences, University of Manchester, Manchester, UK.

Kay Metcalfe (K)

Manchester Centre for Genomic Medicine, St Mary's Hospital, Manchester University Hospitals NHS Foundation Trust, Manchester Academic Health Sciences Centre, Manchester, UK.
Division of Evolution and Genomic Sciences, School of Biological Sciences, University of Manchester, Manchester, UK.

Suresh Somarathi (S)

Manchester Centre for Genomic Medicine, St Mary's Hospital, Manchester University Hospitals NHS Foundation Trust, Manchester Academic Health Sciences Centre, Manchester, UK.

Marc Tischkowitz (M)

Academic Department of Medical Genetics, Cambridge University Hospitals NHS Foundation Trust, University of Cambridge, Cambridge, UK.

Wafik Mohamed (W)

Oxford Centre for Genomic Medicine, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.

Usha Kini (U)

Oxford Centre for Genomic Medicine, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.

Shane McKee (S)

Northern Ireland Regional Genetics Centre, Belfast Health and Social Care Trust, Belfast City Hospital, Belfast, UK.

Laura Yates (L)

West of Scotland Regional Genetics Service, NHS Greater Glasgow and Clyde, Institute of Medical Genetics, Yorkhill Hospital, Glasgow, UK.
KwaZulu-Natal Research and Innovation Sequencing Platform (KRISP), University of KwaZulu-Natal, Durban, South Africa.

Marta Bertoli (M)

West of Scotland Regional Genetics Service, NHS Greater Glasgow and Clyde, Institute of Medical Genetics, Yorkhill Hospital, Glasgow, UK.

Sally Ann Lynch (SA)

Department of Clinical Genetics, Temple Street Children's Hospital, Dublin, Ireland.

Susan Holder (S)

North West Thames Regional Genetics Service, London, UK.
Wellcome Sanger Institute, Cambridge, UK.

Siddharth Banka (S)

Manchester Centre for Genomic Medicine, St Mary's Hospital, Manchester University Hospitals NHS Foundation Trust, Manchester Academic Health Sciences Centre, Manchester, UK.
Division of Evolution and Genomic Sciences, School of Biological Sciences, University of Manchester, Manchester, UK.

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Classifications MeSH