De novo mutations of


Journal

Journal of medical genetics
ISSN: 1468-6244
Titre abrégé: J Med Genet
Pays: England
ID NLM: 2985087R

Informations de publication

Date de publication:
11 2021
Historique:
received: 08 05 2020
revised: 02 08 2020
accepted: 06 08 2020
pubmed: 16 9 2020
medline: 22 2 2022
entrez: 15 9 2020
Statut: ppublish

Résumé

Arthrogryposis multiplex congenita (AMC) is the direct consequence of reduced fetal movements. AMC includes a large spectrum of diseases which result from variants in genes encoding components required for the formation or the function of the neuromuscular system. AMC may also result from central nervous involvement. Whole-exome sequencing was performed from DNA of the index case of AMC families. Heterozygous missense variants in AMC was diagnosed from the second trimester of pregnancy in the three patients. One of them developed drug-resistant epileptic seizures from birth. We showed that We show for the first time that

Sections du résumé

BACKGROUND
Arthrogryposis multiplex congenita (AMC) is the direct consequence of reduced fetal movements. AMC includes a large spectrum of diseases which result from variants in genes encoding components required for the formation or the function of the neuromuscular system. AMC may also result from central nervous involvement.
METHODS
Whole-exome sequencing was performed from DNA of the index case of AMC families. Heterozygous missense variants in
RESULTS
AMC was diagnosed from the second trimester of pregnancy in the three patients. One of them developed drug-resistant epileptic seizures from birth. We showed that
CONCLUSION
We show for the first time that

Identifiants

pubmed: 32928894
pii: jmedgenet-2020-107166
doi: 10.1136/jmedgenet-2020-107166
pmc: PMC8551978
doi:

Substances chimiques

NAV1.1 Voltage-Gated Sodium Channel 0
SCN1A protein, human 0

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

737-742

Informations de copyright

© Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

Competing interests: None declared.

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Auteurs

Dana Jaber (D)

Institut National de la Santé et de la Recherche Médicale (Inserm), UMR-1195, Université Paris Saclay, Le Kremlin Bicêtre, 94276, France.

Cyril Gitiaux (C)

Service de Neurophysiologie Clinique, Centre de référence des maladies neuromusculaires, Hôpital Necker Enfants Malades, Assistance Publique-Hôpitaux de Paris, Université de Paris, Paris, 75015, France.

Sophie Blesson (S)

Service de Génétique, Unité de Génétique Clinique, CHRU de Tours, Hôpital Bretonneau, Tours, 37044, France.

Florent Marguet (F)

Laboratoire de Pathologie, CHU de Rouen and Normandie Univ, Rouen, 76000, France.
INSERM, Laboratoire NeoVasc ERI28, Rouen, 76000, France.

David Buard (D)

Institut National de la Santé et de la Recherche Médicale (Inserm), UMR-1195, Université Paris Saclay, Le Kremlin Bicêtre, 94276, France.

Maritzaida Varela Salgado (M)

Institut National de la Santé et de la Recherche Médicale (Inserm), UMR-1195, Université Paris Saclay, Le Kremlin Bicêtre, 94276, France.

Anna Kaminska (A)

Neurophysiology Department, Necker-Enfants Malades Hospital, Assistance publique-Hôpitaux de Paris, Paris, 75015, France.

Julien Saada (J)

Département d'Obstétrique et Gynécologie, Hôpital Antoine-Béclère, Assistance publique-Hôpitaux de Paris, Clamart, 92140, France.

Catherine Fallet-Bianco (C)

Département de Pathologie, CHU Sainte-Justine, Université de Montréal, Quebec, H3T 1C5, Quebec, Canada.

Jelena Martinovic (J)

Unité de Foetopathologie, Hôpital Antoine-Béclère, Assistance publique-Hôpitaux de Paris, Clamart, 92140, France.

Annie Laquerriere (A)

Laboratoire de Pathologie, CHU de Rouen and Normandie Univ, Rouen, 76000, France.
INSERM, Laboratoire NeoVasc ERI28, Rouen, 76000, France.

Judith Melki (J)

Institut National de la Santé et de la Recherche Médicale (Inserm), UMR-1195, Université Paris Saclay, Le Kremlin Bicêtre, 94276, France judith.melki@inserm.fr.
Unité de Génétique Médicale, Centre de référence des anomalies du développement et syndromes malformatifs d'Île-de-France, Assistance publique-Hôpitaux de Paris, Le Kremlin Bicêtre, 94276, France.

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