Fibrillar form of α-synuclein-specific scFv antibody inhibits α-synuclein seeds induced aggregation and toxicity

<p>Synucleinopathies including Parkinson’s disease (PD), dementia with Lewy bodies (DLB), and multiple system atrophy (MSA) are characterized by pathological accumulation of α-synuclein (α-syn). Amongst the various approaches attempting to tackle the pathological features of synucleinopathies,...

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Main Author: Vijay Gupta (209146) (author)
Other Authors: Safa Salim (9186786) (author), Issam Hmila (9239059) (author), Nishant N. Vaikath (2901785) (author), Indulekha P. Sudhakaran (9611154) (author), Simona S. Ghanem (12267227) (author), Nour K. Majbour (8809316) (author), Sara A. Abdulla (13902015) (author), Mohamed M. Emara (9913215) (author), Houari B. Abdesselem (14152827) (author), Tamas Lukacsovich (23182) (author), Daniel Erskine (3471959) (author), Omar M. A. El-Agnaf (8809331) (author)
Published: 2020
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_version_ 1864513512968028160
author Vijay Gupta (209146)
author2 Safa Salim (9186786)
Issam Hmila (9239059)
Nishant N. Vaikath (2901785)
Indulekha P. Sudhakaran (9611154)
Simona S. Ghanem (12267227)
Nour K. Majbour (8809316)
Sara A. Abdulla (13902015)
Mohamed M. Emara (9913215)
Houari B. Abdesselem (14152827)
Tamas Lukacsovich (23182)
Daniel Erskine (3471959)
Omar M. A. El-Agnaf (8809331)
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author_facet Vijay Gupta (209146)
Safa Salim (9186786)
Issam Hmila (9239059)
Nishant N. Vaikath (2901785)
Indulekha P. Sudhakaran (9611154)
Simona S. Ghanem (12267227)
Nour K. Majbour (8809316)
Sara A. Abdulla (13902015)
Mohamed M. Emara (9913215)
Houari B. Abdesselem (14152827)
Tamas Lukacsovich (23182)
Daniel Erskine (3471959)
Omar M. A. El-Agnaf (8809331)
author_role author
dc.creator.none.fl_str_mv Vijay Gupta (209146)
Safa Salim (9186786)
Issam Hmila (9239059)
Nishant N. Vaikath (2901785)
Indulekha P. Sudhakaran (9611154)
Simona S. Ghanem (12267227)
Nour K. Majbour (8809316)
Sara A. Abdulla (13902015)
Mohamed M. Emara (9913215)
Houari B. Abdesselem (14152827)
Tamas Lukacsovich (23182)
Daniel Erskine (3471959)
Omar M. A. El-Agnaf (8809331)
dc.date.none.fl_str_mv 2020-05-18T03:00:00Z
dc.identifier.none.fl_str_mv 10.1038/s41598-020-65035-8
dc.relation.none.fl_str_mv https://figshare.com/articles/journal_contribution/Fibrillar_form_of_-synuclein-specific_scFv_antibody_inhibits_-synuclein_seeds_induced_aggregation_and_toxicity/21598173
dc.rights.none.fl_str_mv CC BY 4.0
info:eu-repo/semantics/openAccess
dc.subject.none.fl_str_mv Biological sciences
Biochemistry and cell biology
Biomedical and clinical sciences
Neurosciences
Parkinson's disease
Dementia with Lewy bodies
Multiple system atrophy
Neurodegeneration
Immunotherapy
Antibody engineering
Protein purification
dc.title.none.fl_str_mv Fibrillar form of α-synuclein-specific scFv antibody inhibits α-synuclein seeds induced aggregation and toxicity
dc.type.none.fl_str_mv Text
Journal contribution
info:eu-repo/semantics/publishedVersion
text
contribution to journal
description <p>Synucleinopathies including Parkinson’s disease (PD), dementia with Lewy bodies (DLB), and multiple system atrophy (MSA) are characterized by pathological accumulation of α-synuclein (α-syn). Amongst the various approaches attempting to tackle the pathological features of synucleinopathies, antibody-based immunotherapy holds much promise. However, the large size of antibodies and corresponding difficulty in crossing the blood-brain barrier has limited development in this area. To overcome this issue, we engineered single-chain variable fragments (scFvs) against fibrillar α-syn, a putative disease-relevant form of α-syn. The purified scFvs showed specific activity towards α-syn fibrils and oligomers in comparison to monomers and recognized intracellular inclusions in human post-mortem brain tissue of Lewy body disease cases, but not aged controls. In vitro studies indicated scFvs inhibit the seeding of α-syn aggregation in a time-dependent manner, decreased α-syn seed-induced toxicity in a cell model of PD, and reduced the production of insoluble α-syn phosphorylated at Ser-129 (pS129-α-syn). These results suggest that our α-syn fibril-specific scFvs recognize α-syn pathology and can inhibit the aggregation of α-syn in vitro and prevent seeding-dependent toxicity. Therefore, the scFvs described here have considerable potential to be utilized towards immunotherapy in synucleinopathies and may also have applications in ante-mortem imaging modalities.</p><h2>Other Information</h2> <p> Published in: Scientific Reports<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="http://dx.doi.org/10.1038/s41598-020-65035-8" target="_blank">http://dx.doi.org/10.1038/s41598-020-65035-8</a></p>
eu_rights_str_mv openAccess
id Manara2_c6908f2e1672bedecbd970e0e303cab5
identifier_str_mv 10.1038/s41598-020-65035-8
network_acronym_str Manara2
network_name_str Manara2
oai_identifier_str oai:figshare.com:article/21598173
publishDate 2020
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rights_invalid_str_mv CC BY 4.0
spelling Fibrillar form of α-synuclein-specific scFv antibody inhibits α-synuclein seeds induced aggregation and toxicityVijay Gupta (209146)Safa Salim (9186786)Issam Hmila (9239059)Nishant N. Vaikath (2901785)Indulekha P. Sudhakaran (9611154)Simona S. Ghanem (12267227)Nour K. Majbour (8809316)Sara A. Abdulla (13902015)Mohamed M. Emara (9913215)Houari B. Abdesselem (14152827)Tamas Lukacsovich (23182)Daniel Erskine (3471959)Omar M. A. El-Agnaf (8809331)Biological sciencesBiochemistry and cell biologyBiomedical and clinical sciencesNeurosciencesParkinson's diseaseDementia with Lewy bodiesMultiple system atrophyNeurodegenerationImmunotherapyAntibody engineeringProtein purification<p>Synucleinopathies including Parkinson’s disease (PD), dementia with Lewy bodies (DLB), and multiple system atrophy (MSA) are characterized by pathological accumulation of α-synuclein (α-syn). Amongst the various approaches attempting to tackle the pathological features of synucleinopathies, antibody-based immunotherapy holds much promise. However, the large size of antibodies and corresponding difficulty in crossing the blood-brain barrier has limited development in this area. To overcome this issue, we engineered single-chain variable fragments (scFvs) against fibrillar α-syn, a putative disease-relevant form of α-syn. The purified scFvs showed specific activity towards α-syn fibrils and oligomers in comparison to monomers and recognized intracellular inclusions in human post-mortem brain tissue of Lewy body disease cases, but not aged controls. In vitro studies indicated scFvs inhibit the seeding of α-syn aggregation in a time-dependent manner, decreased α-syn seed-induced toxicity in a cell model of PD, and reduced the production of insoluble α-syn phosphorylated at Ser-129 (pS129-α-syn). These results suggest that our α-syn fibril-specific scFvs recognize α-syn pathology and can inhibit the aggregation of α-syn in vitro and prevent seeding-dependent toxicity. Therefore, the scFvs described here have considerable potential to be utilized towards immunotherapy in synucleinopathies and may also have applications in ante-mortem imaging modalities.</p><h2>Other Information</h2> <p> Published in: Scientific Reports<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="http://dx.doi.org/10.1038/s41598-020-65035-8" target="_blank">http://dx.doi.org/10.1038/s41598-020-65035-8</a></p>2020-05-18T03:00:00ZTextJournal contributioninfo:eu-repo/semantics/publishedVersiontextcontribution to journal10.1038/s41598-020-65035-8https://figshare.com/articles/journal_contribution/Fibrillar_form_of_-synuclein-specific_scFv_antibody_inhibits_-synuclein_seeds_induced_aggregation_and_toxicity/21598173CC BY 4.0info:eu-repo/semantics/openAccessoai:figshare.com:article/215981732020-05-18T03:00:00Z
spellingShingle Fibrillar form of α-synuclein-specific scFv antibody inhibits α-synuclein seeds induced aggregation and toxicity
Vijay Gupta (209146)
Biological sciences
Biochemistry and cell biology
Biomedical and clinical sciences
Neurosciences
Parkinson's disease
Dementia with Lewy bodies
Multiple system atrophy
Neurodegeneration
Immunotherapy
Antibody engineering
Protein purification
status_str publishedVersion
title Fibrillar form of α-synuclein-specific scFv antibody inhibits α-synuclein seeds induced aggregation and toxicity
title_full Fibrillar form of α-synuclein-specific scFv antibody inhibits α-synuclein seeds induced aggregation and toxicity
title_fullStr Fibrillar form of α-synuclein-specific scFv antibody inhibits α-synuclein seeds induced aggregation and toxicity
title_full_unstemmed Fibrillar form of α-synuclein-specific scFv antibody inhibits α-synuclein seeds induced aggregation and toxicity
title_short Fibrillar form of α-synuclein-specific scFv antibody inhibits α-synuclein seeds induced aggregation and toxicity
title_sort Fibrillar form of α-synuclein-specific scFv antibody inhibits α-synuclein seeds induced aggregation and toxicity
topic Biological sciences
Biochemistry and cell biology
Biomedical and clinical sciences
Neurosciences
Parkinson's disease
Dementia with Lewy bodies
Multiple system atrophy
Neurodegeneration
Immunotherapy
Antibody engineering
Protein purification