Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s disease

<p dir="ltr">Accumulation of α-synuclein into toxic oligomers or fibrils is implicated in dopaminergic neurodegeneration in Parkinson’s disease. Here we performed a high-throughput, proteome-wide peptide screen to identify protein-protein interaction inhibitors that reduce α-synuclei...

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Main Author: Satra Nim (11540447) (author)
Other Authors: Darren M. O’Hara (9201761) (author), Carles Corbi-Verge (527968) (author), Albert Perez-Riba (5004785) (author), Kazuko Fujisawa (11692038) (author), Minesh Kapadia (582056) (author), Hien Chau (355945) (author), Federica Albanese (19482364) (author), Grishma Pawar (10666899) (author), Mitchell L. De Snoo (19482367) (author), Sophie G. Ngana (19482370) (author), Jisun Kim (837555) (author), Omar M. A. El-Agnaf (8809331) (author), Enrico Rennella (2083291) (author), Lewis E. Kay (135973) (author), Suneil K. Kalia (9201773) (author), Lorraine V. Kalia (9201770) (author), Philip M. Kim (11540462) (author)
Published: 2023
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author Satra Nim (11540447)
author2 Darren M. O’Hara (9201761)
Carles Corbi-Verge (527968)
Albert Perez-Riba (5004785)
Kazuko Fujisawa (11692038)
Minesh Kapadia (582056)
Hien Chau (355945)
Federica Albanese (19482364)
Grishma Pawar (10666899)
Mitchell L. De Snoo (19482367)
Sophie G. Ngana (19482370)
Jisun Kim (837555)
Omar M. A. El-Agnaf (8809331)
Enrico Rennella (2083291)
Lewis E. Kay (135973)
Suneil K. Kalia (9201773)
Lorraine V. Kalia (9201770)
Philip M. Kim (11540462)
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author_facet Satra Nim (11540447)
Darren M. O’Hara (9201761)
Carles Corbi-Verge (527968)
Albert Perez-Riba (5004785)
Kazuko Fujisawa (11692038)
Minesh Kapadia (582056)
Hien Chau (355945)
Federica Albanese (19482364)
Grishma Pawar (10666899)
Mitchell L. De Snoo (19482367)
Sophie G. Ngana (19482370)
Jisun Kim (837555)
Omar M. A. El-Agnaf (8809331)
Enrico Rennella (2083291)
Lewis E. Kay (135973)
Suneil K. Kalia (9201773)
Lorraine V. Kalia (9201770)
Philip M. Kim (11540462)
author_role author
dc.creator.none.fl_str_mv Satra Nim (11540447)
Darren M. O’Hara (9201761)
Carles Corbi-Verge (527968)
Albert Perez-Riba (5004785)
Kazuko Fujisawa (11692038)
Minesh Kapadia (582056)
Hien Chau (355945)
Federica Albanese (19482364)
Grishma Pawar (10666899)
Mitchell L. De Snoo (19482367)
Sophie G. Ngana (19482370)
Jisun Kim (837555)
Omar M. A. El-Agnaf (8809331)
Enrico Rennella (2083291)
Lewis E. Kay (135973)
Suneil K. Kalia (9201773)
Lorraine V. Kalia (9201770)
Philip M. Kim (11540462)
dc.date.none.fl_str_mv 2023-04-19T09:00:00Z
dc.identifier.none.fl_str_mv 10.1038/s41467-023-37464-2
dc.relation.none.fl_str_mv https://figshare.com/articles/journal_contribution/Disrupting_the_-synuclein-ESCRT_interaction_with_a_peptide_inhibitor_mitigates_neurodegeneration_in_preclinical_models_of_Parkinson_s_disease/26830174
dc.rights.none.fl_str_mv CC BY 4.0
info:eu-repo/semantics/openAccess
dc.subject.none.fl_str_mv Biomedical and clinical sciences
Clinical sciences
Neurosciences
Pharmacology and pharmaceutical sciences
α-Synuclein
Parkinson’s Disease
Toxic Oligomers
Protein-Protein Interaction Inhibitors
CHarged Multivesicular body Protein 2B (CHMP2B)
Preclinical Models
C. elegans
Rat Models
dc.title.none.fl_str_mv Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s disease
dc.type.none.fl_str_mv Text
Journal contribution
info:eu-repo/semantics/publishedVersion
text
contribution to journal
description <p dir="ltr">Accumulation of α-synuclein into toxic oligomers or fibrils is implicated in dopaminergic neurodegeneration in Parkinson’s disease. Here we performed a high-throughput, proteome-wide peptide screen to identify protein-protein interaction inhibitors that reduce α-synuclein oligomer levels and their associated cytotoxicity. We find that the most potent peptide inhibitor disrupts the direct interaction between the C-terminal region of α-synuclein and CHarged Multivesicular body Protein 2B (CHMP2B), a component of the Endosomal Sorting Complex Required for Transport-III (ESCRT-III). We show that α-synuclein impedes endolysosomal activity via this interaction, thereby inhibiting its own degradation. Conversely, the peptide inhibitor restores endolysosomal function and thereby decreases α-synuclein levels in multiple models, including female and male human cells harboring disease-causing α-synuclein mutations. Furthermore, the peptide inhibitor protects dopaminergic neurons from α-synuclein-mediated degeneration in hermaphroditic C. elegans and preclinical Parkinson’s disease models using female rats. Thus, the α-synuclein-CHMP2B interaction is a potential therapeutic target for neurodegenerative disorders.</p><h2>Other Information</h2><p dir="ltr">Published in: Nature Communications<br>License: <a href="https://creativecommons.org/licenses/by/4.0" target="_blank">https://creativecommons.org/licenses/by/4.0</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.1038/s41467-023-37464-2" target="_blank">https://dx.doi.org/10.1038/s41467-023-37464-2</a></p>
eu_rights_str_mv openAccess
id Manara2_693c559b6c9a6a71cb865763319f41a7
identifier_str_mv 10.1038/s41467-023-37464-2
network_acronym_str Manara2
network_name_str Manara2
oai_identifier_str oai:figshare.com:article/26830174
publishDate 2023
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rights_invalid_str_mv CC BY 4.0
spelling Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s diseaseSatra Nim (11540447)Darren M. O’Hara (9201761)Carles Corbi-Verge (527968)Albert Perez-Riba (5004785)Kazuko Fujisawa (11692038)Minesh Kapadia (582056)Hien Chau (355945)Federica Albanese (19482364)Grishma Pawar (10666899)Mitchell L. De Snoo (19482367)Sophie G. Ngana (19482370)Jisun Kim (837555)Omar M. A. El-Agnaf (8809331)Enrico Rennella (2083291)Lewis E. Kay (135973)Suneil K. Kalia (9201773)Lorraine V. Kalia (9201770)Philip M. Kim (11540462)Biomedical and clinical sciencesClinical sciencesNeurosciencesPharmacology and pharmaceutical sciencesα-SynucleinParkinson’s DiseaseToxic OligomersProtein-Protein Interaction InhibitorsCHarged Multivesicular body Protein 2B (CHMP2B)Preclinical ModelsC. elegansRat Models<p dir="ltr">Accumulation of α-synuclein into toxic oligomers or fibrils is implicated in dopaminergic neurodegeneration in Parkinson’s disease. Here we performed a high-throughput, proteome-wide peptide screen to identify protein-protein interaction inhibitors that reduce α-synuclein oligomer levels and their associated cytotoxicity. We find that the most potent peptide inhibitor disrupts the direct interaction between the C-terminal region of α-synuclein and CHarged Multivesicular body Protein 2B (CHMP2B), a component of the Endosomal Sorting Complex Required for Transport-III (ESCRT-III). We show that α-synuclein impedes endolysosomal activity via this interaction, thereby inhibiting its own degradation. Conversely, the peptide inhibitor restores endolysosomal function and thereby decreases α-synuclein levels in multiple models, including female and male human cells harboring disease-causing α-synuclein mutations. Furthermore, the peptide inhibitor protects dopaminergic neurons from α-synuclein-mediated degeneration in hermaphroditic C. elegans and preclinical Parkinson’s disease models using female rats. Thus, the α-synuclein-CHMP2B interaction is a potential therapeutic target for neurodegenerative disorders.</p><h2>Other Information</h2><p dir="ltr">Published in: Nature Communications<br>License: <a href="https://creativecommons.org/licenses/by/4.0" target="_blank">https://creativecommons.org/licenses/by/4.0</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.1038/s41467-023-37464-2" target="_blank">https://dx.doi.org/10.1038/s41467-023-37464-2</a></p>2023-04-19T09:00:00ZTextJournal contributioninfo:eu-repo/semantics/publishedVersiontextcontribution to journal10.1038/s41467-023-37464-2https://figshare.com/articles/journal_contribution/Disrupting_the_-synuclein-ESCRT_interaction_with_a_peptide_inhibitor_mitigates_neurodegeneration_in_preclinical_models_of_Parkinson_s_disease/26830174CC BY 4.0info:eu-repo/semantics/openAccessoai:figshare.com:article/268301742023-04-19T09:00:00Z
spellingShingle Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s disease
Satra Nim (11540447)
Biomedical and clinical sciences
Clinical sciences
Neurosciences
Pharmacology and pharmaceutical sciences
α-Synuclein
Parkinson’s Disease
Toxic Oligomers
Protein-Protein Interaction Inhibitors
CHarged Multivesicular body Protein 2B (CHMP2B)
Preclinical Models
C. elegans
Rat Models
status_str publishedVersion
title Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s disease
title_full Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s disease
title_fullStr Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s disease
title_full_unstemmed Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s disease
title_short Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s disease
title_sort Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s disease
topic Biomedical and clinical sciences
Clinical sciences
Neurosciences
Pharmacology and pharmaceutical sciences
α-Synuclein
Parkinson’s Disease
Toxic Oligomers
Protein-Protein Interaction Inhibitors
CHarged Multivesicular body Protein 2B (CHMP2B)
Preclinical Models
C. elegans
Rat Models