Leigh syndrome associated with TRMU gene mutations.

Acute liver failure Leigh syndrome Mitochondrial disease TRMU

Journal

Molecular genetics and metabolism reports
ISSN: 2214-4269
Titre abrégé: Mol Genet Metab Rep
Pays: United States
ID NLM: 101624422

Informations de publication

Date de publication:
Mar 2021
Historique:
received: 15 09 2020
revised: 19 11 2020
accepted: 24 11 2020
entrez: 28 12 2020
pubmed: 29 12 2020
medline: 29 12 2020
Statut: epublish

Résumé

tRNA 5-methylaminomethyl-2-thiouridylate methyltransferase (TRMU) deficiency causes an early onset potentially reversible acute liver failure, so far reported in less than 30 patients. We describe two new unrelated patients with an acute liver failure and a neuroimaging compatible with Leigh syndrome (LS) due to TRMU deficiency, a combination not previously reported. Our report enlarges the phenotypical spectrum of TRMU disease.

Identifiants

pubmed: 33365252
doi: 10.1016/j.ymgmr.2020.100690
pii: S2214-4269(20)30136-1
pmc: PMC7749400
doi:

Types de publication

Journal Article

Langues

eng

Pagination

100690

Informations de copyright

© 2020 The Authors.

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Auteurs

Júlia Sala-Coromina (J)

Pediatric Neurology Department, Vall d'Hebron University Hospital, Universitat Autónoma de Barcelona, Spain.

Lucía Dougherty-de Miguel (LD)

Pediatric Neurology Department, Vall d'Hebron University Hospital, Universitat Autónoma de Barcelona, Spain.

Javier de Las Heras (J)

Division of Pediatric Metabolism, Cruces University Hospital, Biocruces-Bizkaia Health Research Institute, CIBER-ER; University of the Basque Country (UPV/EHU), Spain.

Amaia Lasa-Aranzasti (A)

Department of Clinical and Molecular Genetics, Vall d'Hebron University Hospital, Universitat Autónoma de Barcelona, Spain.

Elena Garcia-Arumi (E)

Department of Clinical and Molecular Genetics, Vall d'Hebron University Hospital, Universitat Autónoma de Barcelona, Spain.
Research Group on Neuromuscular and Mitochondrial Disorders, Vall d'Hebron Institut de Recerca (VHIR), Vall d'Hebron Hospital Universitari, Barcelona, Spain.
Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Instituto de Salud Carlos III, Barcelona, Spain.

Lidia Carreño (L)

Research Group on Neuromuscular and Mitochondrial Disorders, Vall d'Hebron Institut de Recerca (VHIR), Vall d'Hebron Hospital Universitari, Barcelona, Spain.
Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Instituto de Salud Carlos III, Barcelona, Spain.

Jose Antonio Arranz (JA)

Metabolic Laboratory, Vall d'Hebron University Hospital, Universitat Autónoma de Barcelona, Spain.

Clara Carnicer (C)

Metabolic Laboratory, Vall d'Hebron University Hospital, Universitat Autónoma de Barcelona, Spain.

María Unceta-Suárez (M)

Biochemistry Laboratory (Metabolism Area), Cruces University Hospital, Biocruces-Bizkaia Health Research Institute, CIBER-ER, University of the Basque Country (UPV/EHU), Spain.

Angel Sanchez-Montañez (A)

Pediatric Neuroradiology Department, Vall d'Hebron University Hospital, Universitat Autónoma de Barcelona, Spain.

Laura Gort (L)

Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Instituto de Salud Carlos III, Barcelona, Spain.
Inborn Errors of Metabolism, Biochemistry and Molecular Genetics Department, Hospital Clínic, IDIBAPS, Faculty of Medicine and Health Science-University of Barcelona, Internal Medicine Service-Hospital Clínic of Barcelona, Spain.

Frederic Tort (F)

Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Instituto de Salud Carlos III, Barcelona, Spain.
Inborn Errors of Metabolism, Biochemistry and Molecular Genetics Department, Hospital Clínic, IDIBAPS, Faculty of Medicine and Health Science-University of Barcelona, Internal Medicine Service-Hospital Clínic of Barcelona, Spain.

Mireia Del Toro (M)

Pediatric Neurology Department, Vall d'Hebron University Hospital, Universitat Autónoma de Barcelona, Spain.
Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Instituto de Salud Carlos III, Barcelona, Spain.

Classifications MeSH