A mutation in SLC37A4 causes a dominantly inherited congenital disorder of glycosylation characterized by liver dysfunction.


Journal

American journal of human genetics
ISSN: 1537-6605
Titre abrégé: Am J Hum Genet
Pays: United States
ID NLM: 0370475

Informations de publication

Date de publication:
03 06 2021
Historique:
received: 04 02 2021
accepted: 20 04 2021
pubmed: 9 5 2021
medline: 29 6 2021
entrez: 8 5 2021
Statut: ppublish

Résumé

SLC37A4 encodes an endoplasmic reticulum (ER)-localized multitransmembrane protein required for transporting glucose-6-phosphate (Glc-6P) into the ER. Once transported into the ER, Glc-6P is subsequently hydrolyzed by tissue-specific phosphatases to glucose and inorganic phosphate during times of glucose depletion. Pathogenic variants in SLC37A4 cause an established recessive disorder known as glycogen storage disorder 1b characterized by liver and kidney dysfunction with neutropenia. We report seven individuals who presented with liver dysfunction multifactorial coagulation deficiency and cardiac issues and were heterozygous for the same variant, c.1267C>T (p.Arg423

Identifiants

pubmed: 33964207
pii: S0002-9297(21)00144-0
doi: 10.1016/j.ajhg.2021.04.013
pmc: PMC8206404
pii:
doi:

Substances chimiques

Antiporters 0
Monosaccharide Transport Proteins 0
SLC37A4 protein, human 0

Types de publication

Case Reports Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1040-1052

Subventions

Organisme : NCI NIH HHS
ID : P30 CA030199
Pays : United States
Organisme : NIDDK NIH HHS
ID : R01 DK099551
Pays : United States

Informations de copyright

Copyright © 2021 American Society of Human Genetics. Published by Elsevier Inc. All rights reserved.

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Auteurs

Bobby G Ng (BG)

Human Genetics Program, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA 92037, USA.

Paulina Sosicka (P)

Human Genetics Program, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA 92037, USA.

François Fenaille (F)

Université Paris-Saclay, CEA, INRAE, Département Médicaments et Technologies pour la Santé, MetaboHUB, 91191 Gif sur Yvette, France.

Annie Harroche (A)

Hôpital Necker, Haemophilia Care Centre, 75015 Paris, France.

Sandrine Vuillaumier-Barrot (S)

AP-HP, Hôpital Bichat-Claude Bernard, Biochimie Métabolique et Cellulaire, 75018 Paris, France; INSERM U1149, Université de Paris, 75018 Paris, France.

Mindy Porterfield (M)

Complex Carbohydrate Research Center, Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA 30602, USA.

Zhi-Jie Xia (ZJ)

Human Genetics Program, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA 92037, USA.

Shannon Wagner (S)

Complex Carbohydrate Research Center, Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA 30602, USA.

Michael J Bamshad (MJ)

Department of Pediatrics, University of Washington, Seattle, WA 98195, USA; Department of Genome Sciences, University of Washington, Seattle, WA 98195, USA.

Marie-Christine Vergnes-Boiteux (MC)

Laboratoire d'Hématologie, CHU de Bordeaux, 33604 Pessac, France.

Sophie Cholet (S)

Université Paris-Saclay, CEA, INRAE, Département Médicaments et Technologies pour la Santé, MetaboHUB, 91191 Gif sur Yvette, France.

Stephen Dalton (S)

Center for Molecular Medicine, University of Georgia, Athens, GA 30602, USA; Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA 30602, USA.

Anne Dell (A)

Department of Life Sciences, Imperial College London, SW7 2AZ London, UK.

Thierry Dupré (T)

AP-HP, Hôpital Bichat-Claude Bernard, Biochimie Métabolique et Cellulaire, 75018 Paris, France.

Mathieu Fiore (M)

Laboratoire d'Hématologie, CHU de Bordeaux, 33604 Pessac, France; Centre de Référence des Pathologies Plaquettaires Constitutionnelles, CHU de Bordeaux, 33604 Pessac, France.

Stuart M Haslam (SM)

Department of Life Sciences, Imperial College London, SW7 2AZ London, UK.

Yohann Huguenin (Y)

Centre de Ressources et de Compétence des Maladies Hémorragiques Constitutionnelles, CHU de Bordeaux, 33076 Bordeaux, France.

Tadahiro Kumagai (T)

Complex Carbohydrate Research Center, Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA 30602, USA.

Michael Kulik (M)

Center for Molecular Medicine, University of Georgia, Athens, GA 30602, USA; Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA 30602, USA.

Katherine McGoogan (K)

Medical Director of Hepatology Nemours Children's Specialty Care, Jacksonville, FL 32207, USA.

Caroline Michot (C)

Department of Clinical Genetics and Reference Centre for Constitutional Bone Diseases, INSERM U1163, Université de Paris, Imagine Institute, Necker-Enfants Malades Hospital, AP-HP, 75015, Paris, France.

Deborah A Nickerson (DA)

Department of Genome Sciences, University of Washington, Seattle, WA 98195, USA.

Tiffany Pascreau (T)

Laboratoire d'Hématologie Biologique, AP-HP, Hôpital Necker Enfants Malades, 75015 Paris, France.

Delphine Borgel (D)

Laboratoire d'Hématologie Biologique, AP-HP, Hôpital Necker Enfants Malades, 75015 Paris, France; HITh, UMR_S 1176, INSERM, Université Paris-Saclay, 94270 Le Kremlin-Bicêtre, France.

Kimiyo Raymond (K)

Biochemical Genetics Laboratory, Mayo Clinic College of Medicine, Rochester, MN 55905, USA.

Deepti Warad (D)

Division of Pediatric Hematology Oncology, Mayo Clinic, Rochester, MN 55905, USA.

Heather Flanagan-Steet (H)

JC Self Research Institute, Greenwood Genetic Center, Greenwood, SC 29646, USA.

Richard Steet (R)

JC Self Research Institute, Greenwood Genetic Center, Greenwood, SC 29646, USA.

Michael Tiemeyer (M)

Complex Carbohydrate Research Center, Department of Biochemistry and Molecular Biology, University of Georgia, Athens, GA 30602, USA.

Nathalie Seta (N)

AP-HP, Hôpital Bichat-Claude Bernard, Biochimie Métabolique et Cellulaire, 75018 Paris, France.

Arnaud Bruneel (A)

AP-HP, Hôpital Bichat-Claude Bernard, Biochimie Métabolique et Cellulaire, 75018 Paris, France; INSERM UMR1193, Université Paris-Saclay, 92290 Châtenay-Malabry, France.

Hudson H Freeze (HH)

Human Genetics Program, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA 92037, USA. Electronic address: hudson@sbpdiscovery.org.

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Classifications MeSH