CNC-Center for Neuroscience and Cell Biology, University of Coimbra, 3004-504 Coimbra, Portugal.
Center for Innovative Biomedicine and Biotechnology, University of Coimbra, 3004-504 Coimbra, Portugal.
Cells. 2023 Jul 1;12(13):1763. doi: 10.3390/cells12131763.
Amyotrophic lateral sclerosis (ALS) is a severe and incurable neurodegenerative disease characterized by the progressive death of motor neurons, leading to paralysis and death. It is a rare disease characterized by high patient-to-patient heterogeneity, which makes its study arduous and complex. Extracellular vesicles (EVs) have emerged as important players in the development of ALS. Thus, ALS phenotype-expressing cells can spread their abnormal bioactive cargo through the secretion of EVs, even in distant tissues. Importantly, owing to their nature and composition, EVs' formation and cargo can be exploited for better comprehension of this elusive disease and identification of novel biomarkers, as well as for potential therapeutic applications, such as those based on stem cell-derived exosomes. This review highlights recent advances in the identification of the role of EVs in ALS etiopathology and how EVs can be promising new therapeutic strategies.
肌萎缩侧索硬化症（ALS）是一种严重且无法治愈的神经退行性疾病，其特征是运动神经元进行性死亡，导致瘫痪和死亡。这是一种罕见的疾病，其特点是患者之间存在高度异质性，这使得对其进行研究变得艰巨而复杂。细胞外囊泡（EVs）已成为 ALS 发展的重要参与者。因此，表达 ALS 表型的细胞可以通过 EV 的分泌来传播其异常的生物活性货物，即使在远处的组织中也是如此。重要的是，由于其性质和组成，EV 的形成和货物可以被利用来更好地理解这种难以捉摸的疾病，并识别新的生物标志物，以及用于潜在的治疗应用，例如基于干细胞衍生的外泌体。这篇综述强调了细胞外囊泡在 ALS 发病机制中的作用的最新进展，以及细胞外囊泡如何成为有前途的新治疗策略。
