Pathogenic


Journal

Neurology
ISSN: 1526-632X
Titre abrégé: Neurology
Pays: United States
ID NLM: 0401060

Informations de publication

Date de publication:
11 07 2023
Historique:
received: 10 10 2022
accepted: 26 01 2023
pmc-release: 11 07 2024
medline: 12 7 2023
pubmed: 5 4 2023
entrez: 4 4 2023
Statut: ppublish

Résumé

To describe a child meeting diagnostic criteria for tuberous sclerosis complex (TSC) carrying a pathogenic somatic variant in We present the clinical and imaging findings in a child presenting with drug-resistant focal seizures and multiple cortical tubers, a subependymal giant cell astrocytoma and multiple subependymal nodules in 1 cerebral hemisphere. Targeted panel sequencing and exome sequencing were performed on genomic DNA derived from blood and resected tuber tissue. The child satisfied clinical diagnostic criteria for TSC, having 3 major features, only 2 of which are required for diagnosis. Genetic testing did not identify pathogenic variants or copy number variations in RHEB is a partner of the TSC1/2 complex in the mechanistic target of rapamycin pathway. Somatic variants in

Identifiants

pubmed: 37015817
pii: WNL.0000000000207177
doi: 10.1212/WNL.0000000000207177
pmc: PMC10351555
doi:

Substances chimiques

Tumor Suppressor Proteins 0
Tuberous Sclerosis Complex 1 Protein 0
Tuberous Sclerosis Complex 2 Protein 0
RHEB protein, human 0
Ras Homolog Enriched in Brain Protein 0

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

78-82

Informations de copyright

© 2023 American Academy of Neurology.

Références

Gigascience. 2018 Oct 1;7(10):
pubmed: 30192941
Clin EEG Neurosci. 2018 May;49(3):187-191
pubmed: 28762286
Hum Genet. 2010 Mar;127(5):573-82
pubmed: 20165957
Cell Rep. 2017 Dec 26;21(13):3754-3766
pubmed: 29281825
Pediatr Neurol. 2021 Oct;123:50-66
pubmed: 34399110
Exp Mol Med. 2019 Jul 23;51(7):1-11
pubmed: 31337748
Nat Cell Biol. 2003 Jun;5(6):578-81
pubmed: 12771962
Neurology. 2020 Nov 3;95(18):e2542-e2551
pubmed: 32847954
Am J Hum Genet. 2017 Mar 2;100(3):454-472
pubmed: 28215400
PLoS Biol. 2021 May 26;19(5):e3001279
pubmed: 34038402
Acta Neuropathol. 2019 Dec;138(6):885-900
pubmed: 31444548
Eur J Hum Genet. 2005 Jun;13(6):695-6
pubmed: 15912141
Ann Clin Transl Neurol. 2021 Feb;8(2):485-490
pubmed: 33434304
PLoS Genet. 2015 Nov 05;11(11):e1005637
pubmed: 26540169
Am J Hum Genet. 2001 Jan;68(1):64-80
pubmed: 11112665

Auteurs

Wei Shern Lee (WS)

From the Bruce Lefroy Centre, Murdoch Children's Research Institute (W.S.L., S.S., P.J.L.); Department of Paediatrics (W.S.L., E.M-L., S.S., W.M., A.S.H., P.J.L., R.J.L.), The University of Melbourne; Department of Neurology (E.M-L., A.S.H., R.J.L.), The Royal Children's Hospital; Department of Anatomical Pathology (C.D.A.), The Royal Children's Hospital; Department of Neurosurgery (W.M.), The Royal Children's Hospital, Parkville, Australia.

Emma Macdonald-Laurs (E)

From the Bruce Lefroy Centre, Murdoch Children's Research Institute (W.S.L., S.S., P.J.L.); Department of Paediatrics (W.S.L., E.M-L., S.S., W.M., A.S.H., P.J.L., R.J.L.), The University of Melbourne; Department of Neurology (E.M-L., A.S.H., R.J.L.), The Royal Children's Hospital; Department of Anatomical Pathology (C.D.A.), The Royal Children's Hospital; Department of Neurosurgery (W.M.), The Royal Children's Hospital, Parkville, Australia.

Sarah Stephenson (S)

From the Bruce Lefroy Centre, Murdoch Children's Research Institute (W.S.L., S.S., P.J.L.); Department of Paediatrics (W.S.L., E.M-L., S.S., W.M., A.S.H., P.J.L., R.J.L.), The University of Melbourne; Department of Neurology (E.M-L., A.S.H., R.J.L.), The Royal Children's Hospital; Department of Anatomical Pathology (C.D.A.), The Royal Children's Hospital; Department of Neurosurgery (W.M.), The Royal Children's Hospital, Parkville, Australia.

Colleen D'Arcy (C)

From the Bruce Lefroy Centre, Murdoch Children's Research Institute (W.S.L., S.S., P.J.L.); Department of Paediatrics (W.S.L., E.M-L., S.S., W.M., A.S.H., P.J.L., R.J.L.), The University of Melbourne; Department of Neurology (E.M-L., A.S.H., R.J.L.), The Royal Children's Hospital; Department of Anatomical Pathology (C.D.A.), The Royal Children's Hospital; Department of Neurosurgery (W.M.), The Royal Children's Hospital, Parkville, Australia.

Wirginia Maixner (W)

From the Bruce Lefroy Centre, Murdoch Children's Research Institute (W.S.L., S.S., P.J.L.); Department of Paediatrics (W.S.L., E.M-L., S.S., W.M., A.S.H., P.J.L., R.J.L.), The University of Melbourne; Department of Neurology (E.M-L., A.S.H., R.J.L.), The Royal Children's Hospital; Department of Anatomical Pathology (C.D.A.), The Royal Children's Hospital; Department of Neurosurgery (W.M.), The Royal Children's Hospital, Parkville, Australia.

A Simon Harvey (AS)

From the Bruce Lefroy Centre, Murdoch Children's Research Institute (W.S.L., S.S., P.J.L.); Department of Paediatrics (W.S.L., E.M-L., S.S., W.M., A.S.H., P.J.L., R.J.L.), The University of Melbourne; Department of Neurology (E.M-L., A.S.H., R.J.L.), The Royal Children's Hospital; Department of Anatomical Pathology (C.D.A.), The Royal Children's Hospital; Department of Neurosurgery (W.M.), The Royal Children's Hospital, Parkville, Australia.

Paul J Lockhart (PJ)

From the Bruce Lefroy Centre, Murdoch Children's Research Institute (W.S.L., S.S., P.J.L.); Department of Paediatrics (W.S.L., E.M-L., S.S., W.M., A.S.H., P.J.L., R.J.L.), The University of Melbourne; Department of Neurology (E.M-L., A.S.H., R.J.L.), The Royal Children's Hospital; Department of Anatomical Pathology (C.D.A.), The Royal Children's Hospital; Department of Neurosurgery (W.M.), The Royal Children's Hospital, Parkville, Australia.

Richard J Leventer (RJ)

From the Bruce Lefroy Centre, Murdoch Children's Research Institute (W.S.L., S.S., P.J.L.); Department of Paediatrics (W.S.L., E.M-L., S.S., W.M., A.S.H., P.J.L., R.J.L.), The University of Melbourne; Department of Neurology (E.M-L., A.S.H., R.J.L.), The Royal Children's Hospital; Department of Anatomical Pathology (C.D.A.), The Royal Children's Hospital; Department of Neurosurgery (W.M.), The Royal Children's Hospital, Parkville, Australia. richard.leventer@rch.org.au.

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