Discovery of small-molecule positive allosteric modulators of Parkin E3 ligase.

Biochemistry Biochemistry Applications Chemistry

Journal

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

Informations de publication

Date de publication:
21 Jan 2022
Historique:
received: 16 08 2021
revised: 03 11 2021
accepted: 10 12 2021
entrez: 13 1 2022
pubmed: 14 1 2022
medline: 14 1 2022
Statut: epublish

Résumé

Pharmacological activation of the E3 ligase Parkin represents a rational therapeutic intervention for the treatment of Parkinson's disease. Here we identify several compounds that enhance the activity of wildtype Parkin in the presence of phospho-ubiquitin and act as positive allosteric modulators (PAMs). While these compounds activate Parkin in a series of biochemical assays, they do not act by thermally destabilizing Parkin and fail to enhance the Parkin translocation rate to mitochondria or to enact mitophagy in cell-based assays. We conclude that in the context of the cellular milieu the therapeutic window to pharmacologically activate Parkin is very narrow.

Identifiants

pubmed: 35024585
doi: 10.1016/j.isci.2021.103650
pii: S2589-0042(21)01620-5
pmc: PMC8733272
doi:

Types de publication

Journal Article

Langues

eng

Pagination

103650

Informations de copyright

© 2021 The Author(s).

Déclaration de conflit d'intérêts

All authors are employees and shareholders at Biogen.

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Auteurs

Evgeny Shlevkov (E)

Neurodegenerative Disease Research Unit, Biogen, Cambridge, MA 02142, USA.

Paramasivam Murugan (P)

Biotherapeutics and Medicinal Sciences, Biogen, Cambridge, MA 02142, USA.

Dan Montagna (D)

Neurodegenerative Disease Research Unit, Biogen, Cambridge, MA 02142, USA.

Eric Stefan (E)

Biotherapeutics and Medicinal Sciences, Biogen, Cambridge, MA 02142, USA.

Adelajda Hadzipasic (A)

Biotherapeutics and Medicinal Sciences, Biogen, Cambridge, MA 02142, USA.

James S Harvey (JS)

Biotherapeutics and Medicinal Sciences, Biogen, Cambridge, MA 02142, USA.

P Rajesh Kumar (PR)

Biotherapeutics and Medicinal Sciences, Biogen, Cambridge, MA 02142, USA.

Sonya Entova (S)

Biotherapeutics and Medicinal Sciences, Biogen, Cambridge, MA 02142, USA.

Nupur Bansal (N)

Biotherapeutics and Medicinal Sciences, Biogen, Cambridge, MA 02142, USA.

Shari Bickford (S)

Biotherapeutics and Medicinal Sciences, Biogen, Cambridge, MA 02142, USA.

Lai-Yee Wong (LY)

Biotherapeutics and Medicinal Sciences, Biogen, Cambridge, MA 02142, USA.

Warren D Hirst (WD)

Neurodegenerative Disease Research Unit, Biogen, Cambridge, MA 02142, USA.

Andreas Weihofen (A)

Neurodegenerative Disease Research Unit, Biogen, Cambridge, MA 02142, USA.

Laura F Silvian (LF)

Biotherapeutics and Medicinal Sciences, Biogen, Cambridge, MA 02142, USA.

Classifications MeSH