Small Molecule Binding to Alzheimer Risk Factor CD33 Promotes Aβ Phagocytosis.

Components of the Immune System Molecular Neuroscience Molecular Structure Neuroscience Protein Structure Aspects

Journal

iScience
ISSN: 2589-0042
Titre abrégé: iScience
Pays: United States
ID NLM: 101724038

Informations de publication

Date de publication:
27 Sep 2019
Historique:
received: 08 09 2018
revised: 16 01 2019
accepted: 15 07 2019
pubmed: 2 8 2019
medline: 2 8 2019
entrez: 2 8 2019
Statut: ppublish

Résumé

Polymorphism in the microglial receptor CD33 gene has been linked to late-onset Alzheimer disease (AD), and reduced expression of the CD33 sialic acid-binding domain confers protection. Thus, CD33 inhibition might be an effective therapy against disease progression. Progress toward discovery of selective CD33 inhibitors has been hampered by the absence of an atomic resolution structure. We report here the crystal structures of CD33 alone and bound to a subtype-selective sialic acid mimetic called P22 and use them to identify key binding residues by site-directed mutagenesis and binding assays to reveal the molecular basis for its selectivity toward sialylated glycoproteins and glycolipids. We show that P22, when presented on microparticles, increases uptake of the toxic AD peptide, amyloid-β (Aβ), into microglial cells. Thus, the sialic acid-binding site on CD33 is a promising pharmacophore for developing therapeutics that promote clearance of the Aβ peptide that is thought to cause AD.

Identifiants

pubmed: 31369984
pii: S2589-0042(19)30248-2
doi: 10.1016/j.isci.2019.07.023
pmc: PMC6669322
pii:
doi:

Types de publication

Journal Article

Langues

eng

Pagination

110-118

Informations de copyright

Copyright © 2019 The Author(s). Published by Elsevier Inc. All rights reserved.

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Auteurs

Luke A Miles (LA)

ACRF Rational Drug Discovery Centre, St. Vincent's Institute of Medical Research, Fitzroy, VIC 3056, Australia; Department of Biochemistry and Molecular Biology, Bio21 Molecular Science and Biotechnology Institute, University of Melbourne, Parkville, VIC 3010, Australia. Electronic address: lmiles@svi.edu.au.

Stefan J Hermans (SJ)

ACRF Rational Drug Discovery Centre, St. Vincent's Institute of Medical Research, Fitzroy, VIC 3056, Australia; Department of Biochemistry and Molecular Biology, Bio21 Molecular Science and Biotechnology Institute, University of Melbourne, Parkville, VIC 3010, Australia.

Gabriela A N Crespi (GAN)

ACRF Rational Drug Discovery Centre, St. Vincent's Institute of Medical Research, Fitzroy, VIC 3056, Australia; Department of Biochemistry and Molecular Biology, Bio21 Molecular Science and Biotechnology Institute, University of Melbourne, Parkville, VIC 3010, Australia.

Jonathan H Gooi (JH)

ACRF Rational Drug Discovery Centre, St. Vincent's Institute of Medical Research, Fitzroy, VIC 3056, Australia; Department of Biochemistry and Molecular Biology, Bio21 Molecular Science and Biotechnology Institute, University of Melbourne, Parkville, VIC 3010, Australia.

Larissa Doughty (L)

ACRF Rational Drug Discovery Centre, St. Vincent's Institute of Medical Research, Fitzroy, VIC 3056, Australia; Department of Biochemistry and Molecular Biology, Bio21 Molecular Science and Biotechnology Institute, University of Melbourne, Parkville, VIC 3010, Australia.

Tracy L Nero (TL)

ACRF Rational Drug Discovery Centre, St. Vincent's Institute of Medical Research, Fitzroy, VIC 3056, Australia; Department of Biochemistry and Molecular Biology, Bio21 Molecular Science and Biotechnology Institute, University of Melbourne, Parkville, VIC 3010, Australia.

Jasmina Markulić (J)

ACRF Rational Drug Discovery Centre, St. Vincent's Institute of Medical Research, Fitzroy, VIC 3056, Australia; Department of Biochemistry and Molecular Biology, Bio21 Molecular Science and Biotechnology Institute, University of Melbourne, Parkville, VIC 3010, Australia.

Andreas Ebneth (A)

Janssen Research & Development, a Division of Janssen Pharmaceutica N.V, 2340 Beerse, Belgium.

Berthold Wroblowski (B)

Janssen Research & Development, a Division of Janssen Pharmaceutica N.V, 2340 Beerse, Belgium.

Daniel Oehlrich (D)

Janssen Research & Development, a Division of Janssen Pharmaceutica N.V, 2340 Beerse, Belgium.

Andrés A Trabanco (AA)

Neuroscience Medicinal Chemistry, Janssen Research & Development, 45007 Toledo, Spain.

Marie-Laure Rives (ML)

Molecular and Cellular Pharmacology, Janssen Research & Development, LLC, La Jolla, CA 92121, USA.

Ines Royaux (I)

Janssen Research & Development, a Division of Janssen Pharmaceutica N.V, 2340 Beerse, Belgium.

Nancy C Hancock (NC)

ACRF Rational Drug Discovery Centre, St. Vincent's Institute of Medical Research, Fitzroy, VIC 3056, Australia; Department of Biochemistry and Molecular Biology, Bio21 Molecular Science and Biotechnology Institute, University of Melbourne, Parkville, VIC 3010, Australia.

Michael W Parker (MW)

ACRF Rational Drug Discovery Centre, St. Vincent's Institute of Medical Research, Fitzroy, VIC 3056, Australia; Department of Biochemistry and Molecular Biology, Bio21 Molecular Science and Biotechnology Institute, University of Melbourne, Parkville, VIC 3010, Australia. Electronic address: mparker@svi.edu.au.

Classifications MeSH