Xie Huichao, Huang Xiaoyu, Li Bao, Chen Yongfeng, Niu Haoran, Yu Tong, Yang Shimei, Gao Shuxin, Zeng Yutong, Yang Tianzhi, Kang Yan, Zhang Keda, Ding Pingtian
College of Pharmacy, Shenzhen Technology University, Shenzhen, 518118, China.
Guangdong Key Laboratory for Biomedical Measurements and Ultrasound Imaging, National-Regional Key Technology Engineering Laboratory for Medical Ultrasound, School of Biomedical Engineering, Shenzhen University Medical School, Shenzhen, 518060, China.
Adv Sci (Weinh). 2025 Apr;12(15):e2416265. doi: 10.1002/advs.202416265. Epub 2025 Feb 22.
The core strategies in treating rheumatoid arthritis (RA) now focus on ameliorating the inflammatory microenvironment and reversing macrophage phenotypes within the joint cavity. This study introduces a co-delivery system of integrating nanoenzymes and gene therapeutics sequentially modified with guanidinium-based polymers and macrophage membranes to achieve synergistic therapeutic effects. This co-delivery system is named MACP siTNF-α nanoparticles (NPs). MACP siTNF-α nanoparticles are designed for targeted delivery to the inflamed joint site, where they are preferentially internalized by M1-type macrophages and efficiently evade lysosomal degradation. Subsequently, the co-delivery system operates efficiently via a self-sustaining positive feedback drug release mechanism. The biomimetic nanoplatform reduces reactive oxygen species (ROS) levels and prevents glutathione (GSH) depletion. GSH degrades the polymers to release small interfering RNA (siRNA) and expose the Prussian blue (PB) nanoenzymes, which effectively scavenge ROS and restore GSH levels. This feedback loop significantly enhances the gene silencing capability and ROS scavenging efficiency of the co-delivery system. In summary, MACP siTNF-α NPs can reverse macrophage ecological niche in inflammatory soils through the dual mechanism of efficiently inhibiting the expression of tumor necrosis factor-alpha (TNF-α) the upstream pathway of the inflammatory response, and eliminating ROS, thus realizing efficient treatment of RA.
目前,治疗类风湿性关节炎(RA)的核心策略集中在改善炎症微环境以及逆转关节腔内巨噬细胞的表型。本研究引入了一种共递送系统,该系统将纳米酶与基因疗法整合在一起,并用基于胍的聚合物和巨噬细胞膜进行顺序修饰,以实现协同治疗效果。这种共递送系统被命名为MACP siTNF-α纳米颗粒(NPs)。MACP siTNF-α纳米颗粒被设计用于靶向递送至炎症关节部位,在那里它们优先被M1型巨噬细胞内化,并有效逃避溶酶体降解。随后,该共递送系统通过一种自我维持的正反馈药物释放机制高效运行。这种仿生纳米平台降低了活性氧(ROS)水平,并防止谷胱甘肽(GSH)耗竭。GSH降解聚合物以释放小干扰RNA(siRNA)并暴露普鲁士蓝(PB)纳米酶,后者可有效清除ROS并恢复GSH水平。这种反馈回路显著增强了共递送系统的基因沉默能力和ROS清除效率。总之,MACP siTNF-α NPs可以通过有效抑制炎症反应上游途径的肿瘤坏死因子-α(TNF-α)表达以及清除ROS的双重机制,逆转炎症环境中的巨噬细胞生态位,从而实现对RA的有效治疗。