Bellingacci Laura, Sciaccaluga Miriam, Megaro Alfredo, Cardinale Antonella, Canonichesi Jacopo, De Carluccio Maria, Mastrantonio Roberta, Costa Cinzia, Di Filippo Massimiliano, Usiello Alessandro, Viscomi Maria Teresa, Calabresi Paolo, Tozzi Alessandro
Physiology and Biochemistry Section, Department of Medicine and Surgery, University of Perugia, Perugia, Italy.
Fondazione Malattie Rare Mauro Baschirotto BIRD Onlus, Longare (VI), Italy.
NPJ Parkinsons Dis. 2025 Jul 29;11(1):220. doi: 10.1038/s41531-025-01075-z.
In synucleinopathies, α-synuclein oligomers (OSyn) appear to be associated with neurodegeneration, neurotoxicity, and proinflammatory responses, even at low concentrations, suggesting their pivotal role in the pathogenesis of Parkinson's disease (PD). We utilized a rat model of synucleinopathy induced by intrastriatal injection of OSyn, aiming to elucidate events preceding the formation of fibrillary α-syn aggregates. Electrophysiological assessments and behavioral assays revealed several early alterations in OSyn rats, evident as early as 12 weeks post-OSyn injection. These included mild and variable reduction of motor activity, anxiety-like behavior, impaired bidirectional striatal long-term synaptic plasticity, and diminished spontaneous excitatory neurotransmission in the striatum. Furthermore, p-α-syn aggregates were detected in the cortex but not in the substantia nigra (SN). Confocal microscopy analysis revealed reduced vesicular glutamate transporter 1 (VGluT1) expression at striatal glutamatergic terminals. Chronic administration of the ampakine Tulrampator to OSyn animals prevented impairment of long-term depression (LTD), spontaneous striatal neurotransmission, and VGluT1 levels. Tulrampator also ameliorated the anxiety-related behavioral phenotype, albeit without attenuating motor deficits, demonstrating its efficacy in mitigating early synaptic and emotional deficits induced by OSyn. These findings provide a basis for a novel drug treatment strategy aimed at mitigating or delaying early damage at cortico-striatal terminals induced by OSyn, thereby counteracting the pathophysiological processes underlying the onset of early non-motor symptoms in PD.
在突触核蛋白病中,α-突触核蛋白寡聚体(OSyn)似乎与神经退行性变、神经毒性和促炎反应有关,即使在低浓度时也是如此,这表明它们在帕金森病(PD)发病机制中起关键作用。我们利用通过纹状体内注射OSyn诱导的突触核蛋白病大鼠模型,旨在阐明纤维状α-突触核蛋白聚集体形成之前的事件。电生理评估和行为分析揭示了OSyn大鼠的几种早期改变,早在注射OSyn后12周就很明显。这些改变包括运动活动轻度且可变的减少、焦虑样行为、双向纹状体长期突触可塑性受损以及纹状体中自发兴奋性神经传递减弱。此外,在皮质中检测到p-α-突触核蛋白聚集体,但在黑质(SN)中未检测到。共聚焦显微镜分析显示纹状体谷氨酸能终末的囊泡谷氨酸转运体1(VGluT1)表达减少。对OSyn动物长期给予安帕金Tulrampator可预防长期抑郁(LTD)、纹状体自发神经传递和VGluT1水平的损害。Tulrampator还改善了与焦虑相关的行为表型,尽管没有减轻运动缺陷,证明了其在减轻OSyn诱导的早期突触和情绪缺陷方面的功效。这些发现为一种新的药物治疗策略提供了基础,该策略旨在减轻或延迟OSyn诱导的皮质-纹状体终末的早期损伤,从而对抗PD早期非运动症状发作的病理生理过程。