New Perspectives in Dried Blood Spot Biomarkers for Lysosomal Storage Diseases.

Fabry disease Gaucher disease Niemann–Pick C disease biomarker dried blood spot glycosphingolipid mucopolysaccharidoses

Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
15 Jun 2023
Historique:
received: 01 05 2023
revised: 05 06 2023
accepted: 09 06 2023
medline: 29 6 2023
pubmed: 28 6 2023
entrez: 28 6 2023
Statut: epublish

Résumé

Dried blood spots (DBSs) biomarkers are convenient for monitoring for specific lysosomal storage diseases (LSDs), but they could have relevance for other LSDs. To determine the specificity and utility of glycosphingolipidoses biomarkers against other LSDs, we applied a multiplexed lipid liquid chromatography tandem mass spectrometry assay to a DBS cohort of healthy controls (

Identifiants

pubmed: 37373322
pii: ijms241210177
doi: 10.3390/ijms241210177
pmc: PMC10299042
pii:
doi:

Substances chimiques

Glucosylceramides 0
Biomarkers 0
Protein Isoforms 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : Great Ormond Street Biomedical Research Centre
ID : unknown

Références

J Lipid Res. 2019 Aug;60(8):1410-1424
pubmed: 31201291
Blood. 2011 Oct 20;118(16):e118-27
pubmed: 21868580
Am J Hematol. 2016 Nov;91(11):1082-1089
pubmed: 27441734
Ann Lab Med. 2013 Jul;33(4):274-8
pubmed: 23826564
Anal Chem. 2017 Dec 19;89(24):13382-13390
pubmed: 29099167
J Biol Chem. 1998 Oct 2;273(40):25680-5
pubmed: 9748235
Clin Biochem. 2021 Jan;87:79-84
pubmed: 33188770
J Lipid Res. 2016 Mar;57(3):451-63
pubmed: 26724485
Int J Mol Sci. 2017 Oct 20;18(10):
pubmed: 29053611
PLoS One. 2013 Nov 20;8(11):e79732
pubmed: 24278166
Orphanet J Rare Dis. 2015 Jun 17;10:78
pubmed: 26082315
Anal Chem. 2020 Sep 1;92(17):11721-11727
pubmed: 32786498
Neurobiol Dis. 2019 Jul;127:242-252
pubmed: 30872158
Dev Med Child Neurol. 2022 Dec;64(12):1539-1546
pubmed: 35833379
Mol Syndromol. 2023 Feb;14(1):30-34
pubmed: 36777709
Metab Brain Dis. 2019 Oct;34(5):1253-1260
pubmed: 31197681
Diagnostics (Basel). 2020 Mar 13;10(3):
pubmed: 32183018
J Inherit Metab Dis. 2005;28(1):35-48
pubmed: 15702404
Biochim Biophys Acta Mol Basis Dis. 2019 Oct 1;1865(10):2726-2735
pubmed: 31319156
EMBO Mol Med. 2021 Oct 7;13(10):e13742
pubmed: 34411438
J Clin Invest. 1994 Mar;93(3):1288-92
pubmed: 8132768
Anal Biochem. 2017 May 15;525:73-77
pubmed: 28259515
Int J Mol Sci. 2022 Jan 30;23(3):
pubmed: 35163551
Clin Chem Lab Med. 2021 Apr 30;59(9):1516-1526
pubmed: 33915609
Clin Exp Nephrol. 2014 Apr;18(2):269-73
pubmed: 24189976
Hum Mutat. 2016 Jan;37(1):43-51
pubmed: 26415523
J Clin Med. 2021 Apr 13;10(8):
pubmed: 33924567
Acta Paediatr Suppl. 2005 Mar;94(447):51-4; discussion 37-8
pubmed: 15895713
J Clin Med. 2020 Feb 01;9(2):
pubmed: 32024172

Auteurs

Justyna Spiewak (J)

Inborn Errors of Metabolism Section, Genetics & Genomic Medicine Programme, UCL Great Ormond Street Institute of Child Health, University College London, London WC1 1EH, UK.

Ivan Doykov (I)

Inborn Errors of Metabolism Section, Genetics & Genomic Medicine Programme, UCL Great Ormond Street Institute of Child Health, University College London, London WC1 1EH, UK.

Apostolos Papandreou (A)

Inborn Errors of Metabolism Section, Genetics & Genomic Medicine Programme, UCL Great Ormond Street Institute of Child Health, University College London, London WC1 1EH, UK.
Molecular Neurosciences, Developmental Neurosciences Programme, UCL Great Ormond Street Institute of Child Health, University College London, London WC1 1EH, UK.
Department of Neurology, Great Ormond Street Hospital for Children, London WC1N 3JH, UK.

Jenny Hällqvist (J)

Inborn Errors of Metabolism Section, Genetics & Genomic Medicine Programme, UCL Great Ormond Street Institute of Child Health, University College London, London WC1 1EH, UK.

Philippa Mills (P)

Inborn Errors of Metabolism Section, Genetics & Genomic Medicine Programme, UCL Great Ormond Street Institute of Child Health, University College London, London WC1 1EH, UK.

Peter T Clayton (PT)

Inborn Errors of Metabolism Section, Genetics & Genomic Medicine Programme, UCL Great Ormond Street Institute of Child Health, University College London, London WC1 1EH, UK.

Paul Gissen (P)

Inborn Errors of Metabolism Section, Genetics & Genomic Medicine Programme, UCL Great Ormond Street Institute of Child Health, University College London, London WC1 1EH, UK.
Department of Metabolic Medicine, Great Ormond Street Hospital for Children, London WC1N 3JH, UK.

Kevin Mills (K)

Inborn Errors of Metabolism Section, Genetics & Genomic Medicine Programme, UCL Great Ormond Street Institute of Child Health, University College London, London WC1 1EH, UK.

Wendy E Heywood (WE)

Inborn Errors of Metabolism Section, Genetics & Genomic Medicine Programme, UCL Great Ormond Street Institute of Child Health, University College London, London WC1 1EH, UK.

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Classifications MeSH