Guo Qian, Wang Tianying, Qian Christopher, Wang Xinyu
Shanghai 411 Hospital, China RongTong Medical Healthcare Group Co.Ltd. / 411 Hospital, Shanghai University, Shanghai, 200081, China.
Laboratory of Drug Delivery, School of Medicine, Shanghai University, 99 Shangda Road, Shanghai, 200444, China.
Small. 2024 Dec;20(52):e2403980. doi: 10.1002/smll.202403980. Epub 2024 Oct 20.
Current diagnosis and treatment strategies mainly focus on the pathologies of the mid-to-late stage of AD (Alzheimer's disease), with clinical outcomes that are far from ideal. Herein, we developed the ROS (reactive oxygen species)-responsive brain neuronal targeting nanotheranostic platforms that possess the dual-channel fluorescent "turn-on" properties and release drugs in AD neurons in response to ROS, thereby simultaneously facilitating the diagnosis and therapy of early AD. Through the modification of acetylcholine receptor targeting RVG29 peptide, the nanotheranostics penetrated BBB and accumulated into diseased neurons in an intact form, consequently maximizing the diagnostic and therapeutic performance. The anti-oxidative drug baicalein conjugated onto the surface of nanotheranostics via ROS-cleavable boronate ester linkage rapidly released for ROS scavenging, while the encapsulated fluorophores turned on their fluorescence for AD diagnosis upon microenvironment stimuli. This nanotheranostic strategy exhibited highly sensitivity with a ROS detection limit of up to 100 µm and accurately early detection of ROS in 3×Tg AD mice at 6 months of age in vivo. In addition, it could also rescue memory defects, scavenge oxidative stress, attenuate neuroinflammation and enhance neuroprotective effect in 3×Tg AD mice. This work opens up a promising and smart strategy for early diagnosis and therapy in neurodegenerative disease.
目前的诊断和治疗策略主要集中在阿尔茨海默病(AD)中晚期的病理状况上,临床效果远不理想。在此,我们开发了具有活性氧(ROS)响应性的脑神经元靶向纳米诊疗平台,该平台具有双通道荧光“开启”特性,并能响应ROS在AD神经元中释放药物,从而同时促进早期AD的诊断和治疗。通过修饰靶向乙酰胆碱受体的RVG29肽,纳米诊疗剂穿透血脑屏障并完整地聚集到患病神经元中,从而最大限度地提高诊断和治疗性能。通过ROS可裂解的硼酸酯键连接到纳米诊疗剂表面的抗氧化药物黄芩苷可快速释放以清除ROS,而封装的荧光团在微环境刺激下开启荧光用于AD诊断。这种纳米诊疗策略表现出高灵敏度,ROS检测限高达100μm,能够在体内对6月龄的3×Tg AD小鼠的ROS进行准确的早期检测。此外,它还可以挽救3×Tg AD小鼠的记忆缺陷、清除氧化应激、减轻神经炎症并增强神经保护作用。这项工作为神经退行性疾病的早期诊断和治疗开辟了一种有前景的智能策略。