Sun Michael, Miyazawa Kenji, Pendekanti Tejal, Razmi Amaya, Firlar Emre, Yang Stephanie, Shokuhfar Tolou, Li Oliver, Li Wei, Sen Gupta Anirban
Case Western Reserve University, Department of Biomedical Engineering, Cleveland, OH 44106, USA.
Nanoscale. 2020 Nov 7;12(41):21255-21270. doi: 10.1039/d0nr03633a. Epub 2020 Oct 16.
Occlusive thrombosis is a central pathological event in heart attack, stroke, thromboembolism, etc. Therefore, pharmacological thrombolysis or anticoagulation is used for treating these diseases. However, systemic administration of such drugs causes hemorrhagic side-effects. Therefore, there is significant clinical interest in strategies for enhanced drug delivery to clots while minimizing systemic effects. One such strategy is by using drug-carrying nanoparticles surface-decorated with clot-binding ligands. Efforts in this area have focused on binding to singular targets in clots, e.g. platelets, fibrin, collagen, vWF or endothelium. Targeting vWF, collagen or endothelium maybe sub-optimal since in vivo these entities will be rapidly covered by platelets and leukocytes, and thus inaccessible for sufficient nanoparticle binding. In contrast, activated platelets and fibrin are majorly accessible for particle-binding, but their relative distribution in clots is highly heterogeneous. We hypothesized that combination-targeting of 'platelets + fibrin' will render higher clot-binding efficacy of nanoparticles, compared to targeting platelets or fibrin singularly. To test this, we utilized liposomes as model nanoparticles, decorated their surface with platelet-binding peptides (PBP) or fibrin-binding peptides (FBP) or combination (PBP + FBP) at controlled compositions, and evaluated their binding to human blood clots in vitro and in a mouse thrombosis model in vivo. In parallel, we developed a computational model of nanoparticle binding to single versus combination entities in clots. Our studies indicate that combination targeting of 'platelets + fibrin' enhances the clot-anchorage efficacy of nanoparticles while utilizing lower ligand densities, compared to targeting platelets or fibrin only. These findings provide important insights for vascular nanomedicine design.
闭塞性血栓形成是心脏病发作、中风、血栓栓塞等疾病的核心病理事件。因此,药物溶栓或抗凝被用于治疗这些疾病。然而,全身性给药这类药物会引起出血性副作用。因此,在增强药物向血栓的递送同时最小化全身效应的策略方面存在重大临床兴趣。一种这样的策略是使用表面装饰有血栓结合配体的载药纳米颗粒。该领域的努力集中在与血栓中的单一靶点结合,例如血小板、纤维蛋白、胶原蛋白、血管性血友病因子(vWF)或内皮细胞。靶向vWF、胶原蛋白或内皮细胞可能不是最佳选择,因为在体内这些实体将很快被血小板和白细胞覆盖,从而无法实现足够的纳米颗粒结合。相比之下,活化的血小板和纤维蛋白主要可用于颗粒结合,但它们在血栓中的相对分布高度不均一。我们假设,与单独靶向血小板或纤维蛋白相比,“血小板 + 纤维蛋白”的联合靶向将使纳米颗粒具有更高的血栓结合效力。为了验证这一点,我们使用脂质体作为模型纳米颗粒,以可控的组成用血小板结合肽(PBP)或纤维蛋白结合肽(FBP)或二者组合(PBP + FBP)修饰其表面,并在体外和体内小鼠血栓形成模型中评估它们与人血血栓的结合。同时,我们开发了一个纳米颗粒与血栓中单一或联合实体结合的计算模型。我们的研究表明,与仅靶向血小板或纤维蛋白相比,“血小板 + 纤维蛋白”的联合靶向在使用较低配体密度的情况下增强了纳米颗粒的血栓锚定效力。这些发现为血管纳米医学设计提供了重要见解。