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Small-molecule sequestration of amyloid-β as a drug discovery strategy for Alzheimer's disease.

Published version
Peer-reviewed

Type

Article

Change log

Authors

Heller, Gabriella T  ORCID logo  https://orcid.org/0000-0002-5672-0467
Aprile, Francesco A  ORCID logo  https://orcid.org/0000-0002-5040-4420
Michaels, Thomas CT 
Limbocker, Ryan 

Abstract

Disordered proteins are challenging therapeutic targets, and no drug is currently in clinical use that modifies the properties of their monomeric states. Here, we identify a small molecule (10074-G5) capable of binding and sequestering the intrinsically disordered amyloid-β (Aβ) peptide in its monomeric, soluble state. Our analysis reveals that this compound interacts with Aβ and inhibits both the primary and secondary nucleation pathways in its aggregation process. We characterize this interaction using biophysical experiments and integrative structural ensemble determination methods. We observe that this molecule increases the conformational entropy of monomeric Aβ while decreasing its hydrophobic surface area. We also show that it rescues a Caenorhabditis elegans model of Aβ-associated toxicity, consistent with the mechanism of action identified from the in silico and in vitro studies. These results illustrate the strategy of stabilizing the monomeric states of disordered proteins with small molecules to alter their behavior for therapeutic purposes.

Description

Keywords

Alzheimer Disease, Amyloid beta-Peptides, Drug Discovery, Humans, Hydrophobic and Hydrophilic Interactions, Peptide Fragments

Journal Title

Sci Adv

Conference Name

Journal ISSN

2375-2548
2375-2548

Volume Title

6

Publisher

American Association for the Advancement of Science (AAAS)
Sponsorship
Engineering and Physical Sciences Research Council (EP/P020259/1)