Biallelic variants in


Journal

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

Informations de publication

Date de publication:
03 2022
Historique:
received: 28 08 2020
revised: 04 12 2020
accepted: 19 12 2020
pubmed: 6 1 2021
medline: 10 5 2022
entrez: 5 1 2021
Statut: ppublish

Résumé

Next-generation sequencing, combined with international pooling of cases, has impressively enhanced the discovery of genes responsible for Mendelian neurodevelopmental disorders, particularly in individuals affected by clinically undiagnosed diseases. To date, biallelic missense variants in Here, we describe five individuals from four unrelated families with an undiagnosed neurodevelopmental disorder in which we performed exome sequencing, on a combination of trio-based (4 subjects) or single probands (1 subject). We identified five patients from four unrelated families with homozygous Our findings support the role of

Sections du résumé

BACKGROUND
Next-generation sequencing, combined with international pooling of cases, has impressively enhanced the discovery of genes responsible for Mendelian neurodevelopmental disorders, particularly in individuals affected by clinically undiagnosed diseases. To date, biallelic missense variants in
METHODS
Here, we describe five individuals from four unrelated families with an undiagnosed neurodevelopmental disorder in which we performed exome sequencing, on a combination of trio-based (4 subjects) or single probands (1 subject).
RESULTS
We identified five patients from four unrelated families with homozygous
CONCLUSION
Our findings support the role of

Identifiants

pubmed: 33397746
pii: jmedgenet-2020-107430
doi: 10.1136/jmedgenet-2020-107430
doi:

Substances chimiques

Znf526 protein, zebrafish 0

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

262-269

Informations de copyright

© Author(s) (or their employer(s)) 2022. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

Competing interests: None declared.

Auteurs

Maria Lisa Dentici (ML)

Medical Genetics Unit, Academic Department of Pediatrics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy marialisa.dentici@opbg.net.
Genetics and Rare Diseases Research Division, Bambino Gesù Children's Hospital, Rome, Italy.

Viola Alesi (V)

Medical Genetics Laboratory, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Mathieu Quinodoz (M)

Institute of Molecular and Clinical Ophthalmology Basel, Basel, Switzerland.
Department of Ophthalmology, University of Basel, Basel, Switzerland.

Barbara Robens (B)

Department of Neurology, F.M. Kirby Neurobiology Center, Division of Epilepsy and Clinical Neurophysiology and Epilepsy Genetics Program, Boston Children's Hospital, Boston, Massachusetts, USA.
Division of Epilepsy and Clinical Neurophysiology, Boston Children's Hospital, Boston, Massachusetts, USA.

Andrea Guerin (A)

Division of Medical Genetics, Department of Pediatrics, Kingston Health Sciences Centre, Queen's University, Kingston, Ontario, Canada.

Sébastien Lebon (S)

Unit of Pediatric Neurology and Neurorehabilitation Unit, Division of Pediatrics, Department Woman-Mother-Child, Lausanne University Hospital (CHUV), Lausanne, Switzerland.

Annapurna Poduri (A)

Department of Neurology, F.M. Kirby Neurobiology Center, Division of Epilepsy and Clinical Neurophysiology and Epilepsy Genetics Program, Boston Children's Hospital, Boston, Massachusetts, USA.
Division of Epilepsy and Clinical Neurophysiology, Boston Children's Hospital, Boston, Massachusetts, USA.

Lorena Travaglini (L)

Unit of Neuromuscular and Neurodegenerative Diseases, Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Federica Graziola (F)

Neurology Unit, Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Neuroscience department, Tor Vergata University, Rome, Italy.

Alexandra Afenjar (A)

CRMR Déficiences Intellectuelles de Causes Rares, Département de Génétique, Sorbonne Université, APHP, Hôpital Trousseau, Paris, France.

Boris Keren (B)

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

Valerio Licursi (V)

Dept. of Biology and Biotechnology, "Charles Darwin", Sapienza University of Rome, Rome, Italy.

Alessandro Capuano (A)

Neurology Unit, Department of Neuroscience, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Bruno Dallapiccola (B)

Genetics and Rare Diseases Research Division, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

Andrea Superti-Furga (A)

Institute of Molecular and Clinical Ophthalmology Basel, Basel, Switzerland.

Antonio Novelli (A)

Medical Genetics Laboratory, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.

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