Leukocyte and Dried Blood Spot Arylsulfatase A Assay by Tandem Mass Spectrometry.


Journal

Analytical chemistry
ISSN: 1520-6882
Titre abrégé: Anal Chem
Pays: United States
ID NLM: 0370536

Informations de publication

Date de publication:
05 05 2020
Historique:
pubmed: 11 1 2020
medline: 11 2 2021
entrez: 11 1 2020
Statut: ppublish

Résumé

Liquid chromatography-tandem mass spectrometry (LC-MS/MS) assays were developed to measure arylsulfatase A (ARSA) activity in leukocytes and dried blood spots (DBS) using deuterated natural sulfatide substrate. These new assays were highly specific and sensitive. Patients with metachromatic leukodystrophy (MLD) and multiple sulfatase deficiency (MSD) displayed a clear deficit in the enzymatic activity and could be completely distinguished from normal controls. The leukocyte assay reported here will be important for diagnosing MLD and MSD patients and for monitoring the efficacy of therapeutic treatments. ARSA activity was measured in DBS for the first time without an antibody. This new ARSA DBS assay can serve as a second-tier test following the sulfatide measurement in DBS for newborn screening of MLD. This leads to an elimination of most of the false positives identified by the sulfatide assay.

Identifiants

pubmed: 31922725
doi: 10.1021/acs.analchem.9b05274
pmc: PMC7203001
mid: NIHMS1556771
doi:

Substances chimiques

Sulfoglycosphingolipids 0
Cerebroside-Sulfatase EC 3.1.6.8

Types de publication

Journal Article Research Support, N.I.H., Extramural

Langues

eng

Sous-ensembles de citation

IM

Pagination

6341-6348

Subventions

Organisme : NIDDK NIH HHS
ID : R01 DK067859
Pays : United States

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Auteurs

Xinying Hong (X)

Department of Chemistry, University of Washington, Seattle, Washington 98195, United States.

Arun Babu Kumar (AB)

Department of Chemistry, University of Washington, Seattle, Washington 98195, United States.

Jessica Daiker (J)

Department of Chemistry, University of Washington, Seattle, Washington 98195, United States.

Fan Yi (F)

Department of Chemistry, University of Washington, Seattle, Washington 98195, United States.

Martin Sadilek (M)

Department of Chemistry, University of Washington, Seattle, Washington 98195, United States.

Fabiola De Mattia (F)

San Raffaele Telethon Institute for Gene Therapy, IRCCS Ospedale, San Raffaele, Milan,20132, Italy.

Francesca Fumagalli (F)

San Raffaele Telethon Institute for Gene Therapy, IRCCS Ospedale, San Raffaele, Milan,20132, Italy.
Pediatric Immunohematology, IRCCS Ospedale San Raffaele, Milan, 20132, Italy.

Valeria Calbi (V)

San Raffaele Telethon Institute for Gene Therapy, IRCCS Ospedale, San Raffaele, Milan,20132, Italy.
Pediatric Immunohematology, IRCCS Ospedale San Raffaele, Milan, 20132, Italy.

Roberta Damiano (R)

Newborn Screening, Clinical Chemistry, and Pharmacology Lab, Meyer Children's Hospital, Florence, 50139, Italy.

Maria Della Bona (M)

Newborn Screening, Clinical Chemistry, and Pharmacology Lab, Meyer Children's Hospital, Florence, 50139, Italy.

Giancarlo la Marca (G)

Newborn Screening, Clinical Chemistry, and Pharmacology Lab, Meyer Children's Hospital, Florence, 50139, Italy.
Department of Experimental and Clinical Biomedical Sciences, University of Florence, Florence, 50121, Italy.

Adeline L Vanderver (AL)

Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.

Amy T Waldman (AT)

Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.

Laura Adang (L)

Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.
Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States.

Omar Sherbini (O)

Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.

Sarah Woidill (S)

Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States.

Teryn Suhr (T)

MLD Foundation, West Linn, Oregon 97068, United States.

Joanne Kurtzberg (J)

Department of Pediatrics, Duke University, Durham, North Carolina 27705, United States.

Maria L Beltran-Quintero (ML)

University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania 15261, United States.

Maria Escolar (M)

University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania 15261, United States.

Alessandro Aiuti (A)

San Raffaele Telethon Institute for Gene Therapy, IRCCS Ospedale, San Raffaele, Milan,20132, Italy.

Alan Finglas (A)

MSD Action Foundation, Dublin, Ireland.

Amber Olsen (A)

United MSD Foundation, Ocean Springs, Misssissippi 39564, United States.

Michael H Gelb (MH)

Department of Chemistry, University of Washington, Seattle, Washington 98195, United States.
Department of Biochemistry, University of Washington, Seattle, Washington 98195, United States.

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