SCN1A-related epilepsy with recessive inheritance: Two further families.


Journal

European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
ISSN: 1532-2130
Titre abrégé: Eur J Paediatr Neurol
Pays: England
ID NLM: 9715169

Informations de publication

Date de publication:
Jul 2021
Historique:
received: 02 12 2020
revised: 29 04 2021
accepted: 31 05 2021
pubmed: 27 6 2021
medline: 13 10 2021
entrez: 26 6 2021
Statut: ppublish

Résumé

Variants in SCN1A gene, encoding the voltage-gated sodium channel Na We report two novel homozygous SCN1A missense variants in two patients from related parents. Both patients had fever-sensitive epilepsy beginning in the first months of life, followed by afebrile seizures, without severe cognitive impairment. Parents were asymptomatic. Next generation sequencing excluded a pathogenic variant in other genes involved in DEE. Estimation of pathogenicity scores by in-silico tools suggests that the impact of these SCN1A variants is less damaging than that of dominant pathogenic variants. This study provides additional evidence that homozygous variants in SCN1A can cause GEFS+. This recessive inheritance would imply that hypomorphic variants may not necessarily cause epilepsy at the heterozygous state but may decrease the seizure threshold when combined.

Sections du résumé

BACKGROUND BACKGROUND
Variants in SCN1A gene, encoding the voltage-gated sodium channel Na
METHODS AND RESULTS RESULTS
We report two novel homozygous SCN1A missense variants in two patients from related parents. Both patients had fever-sensitive epilepsy beginning in the first months of life, followed by afebrile seizures, without severe cognitive impairment. Parents were asymptomatic. Next generation sequencing excluded a pathogenic variant in other genes involved in DEE. Estimation of pathogenicity scores by in-silico tools suggests that the impact of these SCN1A variants is less damaging than that of dominant pathogenic variants.
CONCLUSION CONCLUSIONS
This study provides additional evidence that homozygous variants in SCN1A can cause GEFS+. This recessive inheritance would imply that hypomorphic variants may not necessarily cause epilepsy at the heterozygous state but may decrease the seizure threshold when combined.

Identifiants

pubmed: 34174751
pii: S1090-3798(21)00122-7
doi: 10.1016/j.ejpn.2021.05.018
pii:
doi:

Substances chimiques

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

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

121-124

Informations de copyright

Copyright © 2021 European Paediatric Neurology Society. Published by Elsevier Ltd. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of competing interest None.

Auteurs

Raffaella Moretti (R)

APHP.Sorbonne Université, Département de Physiologie, Hôpital Trousseau, Paris, France.

Lionel Arnaud (L)

APHP.Sorbonne Université, Département de Génétique, Groupe Hospitalier Pitié Salpêtrière, Paris, France.

Delphine Bouteiller (D)

Sorbonne Université, Institut du Cerveau, ICM, Inserm U1127, CNRS UMR 7225, Paris, France.

Oriane Trouillard (O)

APHP.Sorbonne Université, Département de Génétique, Groupe Hospitalier Pitié Salpêtrière, Paris, France; Sorbonne Université, Institut du Cerveau, ICM, Inserm U1127, CNRS UMR 7225, Paris, France.

Patricia Moreau (P)

APHP.Sorbonne Université, Département de Génétique, Groupe Hospitalier Pitié Salpêtrière, Paris, France.

Julien Buratti (J)

APHP.Sorbonne Université, Département de Génétique, Groupe Hospitalier Pitié Salpêtrière, Paris, France.

Agnès Rastetter (A)

Sorbonne Université, Institut du Cerveau, ICM, Inserm U1127, CNRS UMR 7225, Paris, France.

Boris Keren (B)

APHP.Sorbonne Université, Département de Génétique, Groupe Hospitalier Pitié Salpêtrière, Paris, France.

Vincent Des Portes (V)

Reference Center for Rare Epilepsies CRéER, Lyon University Hospital, F-69677, Bron, France; University Lyon 1, F-69008, Lyon, France.

Joseph Toulouse (J)

Reference Center for Rare Epilepsies CRéER, Lyon University Hospital, F-69677, Bron, France.

Isabelle Gourfinkel-An (I)

APHP.Sorbonne Université, Département de Neurologie, Centre de Référence des Épilepsies Rares, Groupe Hospitalier Pitié Salpêtrière, Paris, France.

Eric Leguern (E)

APHP.Sorbonne Université, Département de Génétique, Groupe Hospitalier Pitié Salpêtrière, Paris, France; Sorbonne Université, Institut du Cerveau, ICM, Inserm U1127, CNRS UMR 7225, Paris, France.

Christel Depienne (C)

Sorbonne Université, Institut du Cerveau, ICM, Inserm U1127, CNRS UMR 7225, Paris, France; Institute of Human Genetics, University Hospital Essen, University of Duisburg-Essen, Essen, Germany.

Cyril Mignot (C)

APHP.Sorbonne Université, Département de Génétique, Groupe Hospitalier Pitié Salpêtrière, Paris, France; Sorbonne Université, Institut du Cerveau, ICM, Inserm U1127, CNRS UMR 7225, Paris, France; Centre de Référence Déficiences Intellectuelles de Causes Rares, GH Pitié-Salpêtrière, Paris, France.

Caroline Nava (C)

APHP.Sorbonne Université, Département de Génétique, Groupe Hospitalier Pitié Salpêtrière, Paris, France; Sorbonne Université, Institut du Cerveau, ICM, Inserm U1127, CNRS UMR 7225, Paris, France. Electronic address: caroline.nava@aphp.fr.

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