Cordeiro Yraima, Freire Maria Heloisa O, Wiecikowski Adalgisa Felippe, do Amaral Mariana Juliani
Faculty of Pharmacy, Universidade Federal do Rio de Janeiro, Av Carlos Chagas Filho 373, bloco B, subsolo Sala 36, Rio de Janeiro, RJ 21941-902 Brazil.
Biophys Rev. 2023 Jun 6;15(4):577-589. doi: 10.1007/s12551-023-01067-4. eCollection 2023 Aug.
Prion diseases are prototype of infectious diseases transmitted by a protein, the prion protein (PrP), and are still not understandable at the molecular level. Heterogenous species of aggregated PrP can be generated from its monomer. α-synuclein (αSyn), related to Parkinson's disease, has also shown a prion-like pathogenic character, and likewise PrP interacts with nucleic acids (NAs), which in turn modulate their aggregation. Recently, our group and others have characterized that NAs and/or RNA-binding proteins (RBPs) modulate recombinant PrP and/or αSyn condensates formation, and uncontrolled condensation might precede pathological aggregation. Tackling abnormal phase separation of neurodegenerative disease-related proteins has been proposed as a promising therapeutic target. Therefore, understanding the mechanism by which polyanions, like NAs, modulate phase transitions intracellularly, is key to assess their role on toxicity promotion and neuronal death. Herein we discuss data on the nucleic acids binding properties and phase separation ability of PrP and αSyn with a special focus on their modulation by NAs and RBPs. Furthermore, we provide insights into condensation of PrP and/or αSyn in the light of non-trivial subcellular locations such as the nuclear and cytosolic environments.
朊病毒疾病是由蛋白质(朊病毒蛋白,PrP)传播的传染病的典型代表,在分子水平上仍难以理解。聚集的PrP可由其单体产生多种异质形式。与帕金森病相关的α-突触核蛋白(αSyn)也表现出类似朊病毒的致病特性,同样,PrP与核酸(NAs)相互作用,进而调节其聚集。最近,我们团队和其他团队已经证实,核酸和/或RNA结合蛋白(RBPs)可调节重组PrP和/或αSyn凝聚物的形成,不受控制的凝聚可能先于病理性聚集。解决神经退行性疾病相关蛋白的异常相分离已被提议作为一个有前景的治疗靶点。因此,了解像核酸这样的多阴离子在细胞内调节相变的机制,是评估它们在促进毒性和神经元死亡中作用的关键。在此,我们讨论关于PrP和αSyn的核酸结合特性和相分离能力的数据,特别关注核酸和RBPs对它们的调节作用。此外,鉴于核和胞质环境等非平凡亚细胞位置,我们对PrP和/或αSyn的凝聚提供了见解。