Department of Chemistry, Bar-Ilan University, Ramat-Gan, 5290002, Israel.
Small. 2024 Aug;20(31):e2311670. doi: 10.1002/smll.202311670. Epub 2024 Mar 10.
Early detection and treatment are crucial for Alzheimer's disease (AD) management. Current diagnostic and therapeutic methods focus on late-stage amyloid fibrils and plaques, overlooking toxic soluble amyloid β oligomers (AβOs) accumulating early in AD. A multifunctional liposome-based platform is designed for early diagnosis and therapy of AD, leveraging a novel self-assembled cyclic d,l-α-peptide (CP-2) that selectively targets AβOs. Biocompatible CP-2 conjugated liposomes (CP-2-LPs) effectively disrupt Aβ aggregation and mitigate Aβ-mediated toxicity in human neuroblastoma cells. In transgenic Caenorhabditis elegans AD models, CP-2-LPs significantly outperformed free CP-2 by improving cognitive and behavioral functions, extending lifespan, and reducing toxic AβO levels. Intravenous injection of fluorescently labeled CP-2-LPs reveals effective blood-brain barrier penetration, with significantly higher brain fluorescence in transgenic mice than WT, enabling precise diagnosis. These findings underscore CP-2-LPs as a valuable tool for early detection and targeted therapy in AD.
早期发现和治疗对阿尔茨海默病(AD)的管理至关重要。目前的诊断和治疗方法侧重于晚期淀粉样纤维和斑块,而忽略了 AD 早期积累的有毒可溶性淀粉样 β 寡聚物(AβOs)。设计了一种基于多功能脂质体的平台,用于 AD 的早期诊断和治疗,利用一种新型自组装的环 dl-α-肽(CP-2),该肽选择性靶向 AβOs。生物相容的 CP-2 缀合的脂质体(CP-2-LPs)可有效破坏 Aβ 聚集,并减轻人神经母细胞瘤细胞中的 Aβ 介导的毒性。在转基因秀丽隐杆线虫 AD 模型中,CP-2-LPs 通过改善认知和行为功能、延长寿命和降低毒性 AβO 水平,显著优于游离 CP-2。荧光标记的 CP-2-LPs 的静脉内注射显示出有效的血脑屏障穿透性,在转基因小鼠中的脑荧光显著高于 WT,从而能够进行精确诊断。这些发现强调了 CP-2-LPs 作为 AD 早期检测和靶向治疗的有价值工具。