Mitochondrial energetic impairment in a patient with late-onset glutaric acidemia Type 2.


Journal

American journal of medical genetics. Part A
ISSN: 1552-4833
Titre abrégé: Am J Med Genet A
Pays: United States
ID NLM: 101235741

Informations de publication

Date de publication:
10 2020
Historique:
received: 16 04 2020
revised: 22 06 2020
accepted: 29 06 2020
pubmed: 18 8 2020
medline: 2 6 2021
entrez: 18 8 2020
Statut: ppublish

Résumé

Glutaric acidemia type 2 (GA2), also called multiple acyl-CoA dehydrogenase deficiency, is an autosomal recessive disorder of fatty acid, amino acid, and choline metabolism resulting in excretion of multiple organic acids and glycine conjugates as well as elevation of various plasma acylcarnitine species (C4-C18). It is caused by mutations in the ETFA, ETFB, or ETFDH genes which are involved in the transfer of electrons from 11 flavin-containing dehydrogenases to Coenzyme Q

Identifiants

pubmed: 32804429
doi: 10.1002/ajmg.a.61786
pmc: PMC8543298
mid: NIHMS1728432
doi:

Substances chimiques

Electron-Transferring Flavoproteins 0
Iron-Sulfur Proteins 0
Ubiquinone 1339-63-5
Acyl-CoA Dehydrogenase, Long-Chain EC 1.3.8.8
ACADVL protein, human EC 1.3.8.9
Oxidoreductases Acting on CH-NH Group Donors EC 1.5.-
electron-transferring-flavoprotein dehydrogenase EC 1.5.5.1
coenzyme Q10 EJ27X76M46

Types de publication

Case Reports Research Support, N.I.H., Extramural

Langues

eng

Sous-ensembles de citation

IM

Pagination

2426-2431

Subventions

Organisme : NIDDK NIH HHS
ID : K08 DK105233
Pays : United States
Organisme : NIDDK NIH HHS
ID : R01 DK078775
Pays : United States
Organisme : NIDDK NIH HHS
ID : K08DK105233
Pays : United States

Informations de copyright

© 2020 Wiley Periodicals LLC.

Références

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Auteurs

Changrui Xiao (C)

National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.

Esteban Astiazaran-Symonds (E)

National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.

Shrabani Basu (S)

Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA.

Monisha Kisling (M)

Rare Disease Institute, Children's National Health System, Washington, District of Columbia, USA.

Fernando Scaglia (F)

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas, USA.
Texas Children's Hospital, Houston, Texas, USA.
BCM-CUHK Center of Medical Genetics, Prince of Wales Hospital, Hong Kong, SAR.

Kimberly A Chapman (KA)

Rare Disease Institute, Children's National Health System, Washington, District of Columbia, USA.

Yudong Wang (Y)

Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA.

Jerry Vockley (J)

Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA.
Department of Human Genetics, University of Pittsburgh Graduate School of Public Health, Pittsburgh, Pennsylvania, USA.
Center for Rare Disease Therapy, UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.

Carlos R Ferreira (CR)

National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.

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