The small molecule -2 inhibits the aggregation and seeded polymerisation of C-terminally truncated α-Synuclein

Protein aggregation, particularly the formation of amyloid fibrils, is associated with numerous human disorders, including Parkinson's disease. This neurodegenerative condition is characterised by the accumulation of α-Synuclein amyloid fibrils within intraneuronal deposits known as Lewy bodies...

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Detalles Bibliográficos
Autores: Peña Díaz, Samuel|||0000-0002-2902-823X, Ventura, Salvador|||0000-0002-9652-6351
Tipo de recurso: artículo
Fecha de publicación:2024
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:307580
Acceso en línea:https://ddd.uab.cat/record/307580
https://dx.doi.org/urn:doi:10.1111/febs.17310
Access Level:acceso abierto
Palabra clave:Inhibition
Oligomer
Protein aggregation
Truncation
α-Synuclein
Descripción
Sumario:Protein aggregation, particularly the formation of amyloid fibrils, is associated with numerous human disorders, including Parkinson's disease. This neurodegenerative condition is characterised by the accumulation of α-Synuclein amyloid fibrils within intraneuronal deposits known as Lewy bodies or neurites. C-terminally truncated forms of α-Synuclein are frequently observed in these inclusions in the brains of patients, and their increased aggregation propensity suggests a role in the disease's pathogenesis. This study demonstrates that the small molecule ZPD-2 acts as a potent inhibitor of both the spontaneous and seeded amyloid polimerisation of C-terminally truncated α-Synuclein by interfering with early aggregation intermediates. This dual activity positions this molecule as a promising candidate for therapeutic development in treating synucleinopathies. The aggregation of α-Synuclein plays a key role in Parkinson's disease. Remarkably, the C-terminal region of this protein contributes to inhibit amyloid formation and its truncation enhances this process. In this work, we demonstrated that the addition of small molecules, ZPD-2 or SynuClean-D, inhibits the aggregation of C-terminally truncated α-Synuclein. Overall, the data suggested different inhibitory mechanisms with ZPD-2 outperforming SC-D by acting at the initial stages of aggregation.