Dementia Research Group, Korea Brain Research Institute (KBRI), Daegu, 41062, South Korea.
Department of Brain & Cognitive Sciences, DGIST, Daegu, 42988, South Korea.
Exp Mol Med. 2020 Oct;52(10):1652-1662. doi: 10.1038/s12276-020-00513-7. Epub 2020 Oct 13.
TAR DNA-binding protein 43 (TDP-43) is a highly conserved nuclear RNA/DNA-binding protein involved in the regulation of RNA processing. The accumulation of TDP-43 aggregates in the central nervous system is a common feature of many neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), and limbic predominant age-related TDP-43 encephalopathy (LATE). Accumulating evidence suggests that prion-like spreading of aberrant protein aggregates composed of tau, amyloid-β, and α-synuclein is involved in the progression of neurodegenerative diseases such as AD and PD. Similar to those of prion-like proteins, pathological aggregates of TDP-43 can be transferred from cell-to-cell in a seed-dependent and self-templating manner. Here, we review clinical and experimental studies supporting the prion-like spreading of misfolded TDP-43 and discuss the molecular mechanisms underlying the propagation of these pathological aggregated proteins. The idea that misfolded TDP-43 spreads in a prion-like manner between cells may guide novel therapeutic strategies for TDP-43-associated neurodegenerative diseases.
TAR DNA 结合蛋白 43(TDP-43)是一种高度保守的核 RNA/DNA 结合蛋白,参与 RNA 加工的调节。TDP-43 聚集物在中枢神经系统中的积累是许多神经退行性疾病的共同特征,如肌萎缩侧索硬化症(ALS)、额颞叶痴呆(FTD)、阿尔茨海默病(AD)和边缘为主的年龄相关性 TDP-43 脑病(LATE)。越来越多的证据表明,由 tau、淀粉样β和α-突触核蛋白组成的异常蛋白聚集的类朊病毒样扩散参与了 AD 和 PD 等神经退行性疾病的进展。与类朊病毒样蛋白类似,TDP-43 的病理性聚集可以以依赖于种子的自我模板方式在细胞间进行传递。在这里,我们综述了支持错误折叠的 TDP-43 类朊病毒样扩散的临床和实验研究,并讨论了这些病理性聚集蛋白传播的分子机制。错误折叠的 TDP-43 以类朊病毒样方式在细胞间传播的观点可能为 TDP-43 相关神经退行性疾病的新型治疗策略提供指导。