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神经退行性疾病:转化药物发现的发病机制与临床前模型

Neurodegenerative Diseases: Pathogenesis and Preclinical Models for Translational Drug Discovery.

作者信息

Fusaro Laura, Bangari Dinesh S, Pasterkamp R Jeroen, Fernández-Ruiz Javier, Youssef Sameh A, Sharma Alok K

机构信息

Denali Therapeutics, South San Francisco, California, USA.

Sanofi, Cambridge, Massachusetts, USA.

出版信息

Toxicol Pathol. 2025 Jun;53(4):364-371. doi: 10.1177/01926233251339105. Epub 2025 May 15.

Abstract

The fourth session of the 2024 European Society of Toxicologic Pathology (ESTP) Congress brought together lectures focused on the use of in vitro and in vivo models to investigate neurodegenerative diseases. Four presentations highlighted various aspects of neurodegenerative diseases including dementia, immune-mediated conditions, and neuromuscular disorders. The session began with an overview of animal models of dementia underscoring their critical role in understanding disease pathogenesis and supporting the development of effective therapeutic drugs. Subsequent presentations investigated immunological self-tolerance in autoimmune neurodegenerative diseases, such as multiple sclerosis and Guillain-Barré syndrome, and the application of in vitro models to study neuromuscular diseases such as amyotrophic lateral sclerosis. The final presentation examined cannabinoid-based therapeutic options for treating neurodegenerative diseases, highlighting their potential in neuroprotection and neurorepair. This session provided valuable insights into the latest research and advancements in neurodegenerative disease modeling and therapy, offering promising directions for improved modeling and therapeutic strategies.

摘要

2024年欧洲毒理病理学会(ESTP)第四届大会汇聚了多场讲座,重点关注使用体外和体内模型来研究神经退行性疾病。四场报告突出了神经退行性疾病的各个方面,包括痴呆症、免疫介导疾病和神经肌肉疾病。会议首先概述了痴呆症的动物模型,强调了它们在理解疾病发病机制和支持有效治疗药物研发方面的关键作用。随后的报告研究了自身免疫性神经退行性疾病(如多发性硬化症和格林-巴利综合征)中的免疫自我耐受性,以及体外模型在研究肌萎缩侧索硬化症等神经肌肉疾病中的应用。最后一场报告探讨了基于大麻素的神经退行性疾病治疗选择,强调了它们在神经保护和神经修复方面的潜力。本次会议为神经退行性疾病建模和治疗的最新研究与进展提供了宝贵见解,为改进建模和治疗策略提供了有前景的方向。

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