Department of Chemical & Biomolecular Engineering, University of Notre Dame, Indiana 46556, USA.
Department of Chemistry & Biochemistry, University of Notre Dame, Indiana 46556, USA.
J Mater Chem B. 2023 Aug 9;11(31):7378-7388. doi: 10.1039/d3tb00850a.
Tauopathies are a class of neurodegenerative diseases resulting in cognitive dysfunction, executive dysfunction, and motor disturbance. The primary pathological feature of tauopathies is the presence of neurofibrillary tangles in the brain composed of tau protein aggregates. Moreover, tau aggregates can spread from neuron to neuron and lead to the propagation of tau pathology. Although numerous small molecules are known to inhibit tau aggregation and block tau cell-to-cell transmission, it is still challenging to use them for therapeutic applications due to poor specificity and low blood-brain barrier (BBB) penetration. Graphene nanoparticles were previously demonstrated to penetrate the BBB and are amenable to functionalization for targeted delivery. Moreover, these nanoscale biomimetic particles can self-assemble or assemble with various biomolecules including proteins. In this paper, we show that graphene quantum dots (GQDs), as graphene nanoparticles, block the seeding activity of tau fibrils by inhibiting the fibrillization of monomeric tau and triggering the disaggregation of tau filaments. This behavior is attributed to electrostatic and π-π stacking interactions of GQDs with tau. Overall, our studies indicate that GQDs with biomimetic properties can efficiently inhibit and disassemble pathological tau aggregates, and thus block tau transmission, which supports their future developments as a potential treatment for tauopathies.
tau 病是一类导致认知功能障碍、执行功能障碍和运动障碍的神经退行性疾病。tau 病的主要病理特征是大脑中存在由 tau 蛋白聚集物组成的神经纤维缠结。此外,tau 聚集物可以从神经元传播到神经元,导致 tau 病理学的传播。尽管已经有许多小分子被证实可以抑制 tau 聚集并阻断 tau 细胞间传递,但由于特异性差和血脑屏障 (BBB) 通透性低,这些小分子在治疗应用中仍然具有挑战性。先前已经证明石墨烯纳米粒子可以穿透 BBB,并且易于功能化以实现靶向递送。此外,这些纳米级仿生粒子可以自组装或与包括蛋白质在内的各种生物分子组装。在本文中,我们表明,作为石墨烯纳米粒子的石墨烯量子点 (GQDs) 通过抑制单体 tau 的纤维化和触发 tau 丝的解聚来阻止 tau 原纤维的成核活性。这种行为归因于 GQDs 与 tau 之间的静电和 π-π 堆积相互作用。总的来说,我们的研究表明,具有仿生特性的 GQDs 可以有效地抑制和拆解病理性 tau 聚集物,从而阻断 tau 传递,这支持了它们作为 tau 病潜在治疗方法的未来发展。