Despite sustained efforts to treat neurodegenerative diseases, little is known at the molecular level to understand and generate novel therapeutic approaches for these malignancies. Therefore, it is not surprising that neurogenerative diseases are among the leading causes of death in the aged population. Neurons require sophisticated cellular mechanisms to maintain proper protein homeostasis. These cells are generally sensitive to loss of gene expression control at the post-transcriptional level. Post-translational control responds to signals that can arise from intracellular processes or environmental factors that can be regulated through RNA-binding proteins. These proteins recognize RNA through one or more RNA-binding domains and form ribonucleoproteins that are critically involved in the regulation of post-transcriptional processes from splicing to the regulation of association of the translation machinery allowing a relatively rapid and precise modulation of the transcriptome. Neurotoxicity is the result of the biological, chemical, or physical interaction of agents with an adverse effect on the structure and function of the central nervous system. The disruption of the proper levels or function of RBPs in neurons and glial cells triggers neurotoxic events that are linked to neurodegenerative diseases such as spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), fragile X syndrome (FXS), and frontotemporal dementia (FTD) among many others. The connection between RBPs and neurodegenerative diseases opens a new landscape for potentially novel therapeutic targets for the intervention of these neurodegenerative pathologies. In this contribution, a summary of the recent findings of the molecular mechanisms involved in the plausible role of RBPs in RNA processing in neurodegenerative disease is discussed.

尽管人们不断努力治疗神经退行性疾病，但在分子水平上对理解和产生这些恶性肿瘤的新治疗方法知之甚少。因此，神经生成性疾病成为老年人口死亡的主要原因之一也就不足为奇了。神经元需要复杂的细胞机制来维持适当的蛋白质稳态。这些细胞通常对转录后水平的基因表达控制的丧失敏感。翻译后控制对细胞内过程或环境因素产生的信号做出反应，这些信号可通过 RNA 结合蛋白进行调节。这些蛋白质通过一个或多个RNA结合结构域识别RNA并形成核糖核蛋白，这些核糖核蛋白关键参与从剪接到翻译机器关联调节的转录后过程的调节，从而允许相对快速和精确地调节转录组。神经毒性是药物生物、化学或物理相互作用的结果，对中枢神经系统的结构和功能产生不利影响。神经元和神经胶质细胞中 RBP 的正常水平或功能受到破坏，会引发与神经退行性疾病相关的神经毒性事件，例如脊髓性肌萎缩症 (SMA)、肌萎缩侧索硬化症 (ALS)、脆性 X 综合征 (FXS) 和额颞叶痴呆（FTD）等等。RBP 与神经退行性疾病之间的联系为干预这些神经退行性疾病的潜在新治疗靶点开辟了新的前景。在这篇文章中，讨论了神经退行性疾病中 RBP 在 RNA 加工中的合理作用所涉及的分子机制的最新发现。