Meng Ran, Li Yixian, Yang Xiyu, Cheng Yunlong, Xu Minjun, Zhou LingLing, Wu Chengqin, Yu Shuai, Huang Wenyi, Wang Tianying, Zhang Qizhi
Key Laboratory of Smart Drug Delivery, Ministry of Education, National Key Laboratory of Advanced Drug Formulations for Overcoming Delivery Barriers, School of Pharmacy, Fudan University, Shanghai, 201203, P. R. China.
Shanxi Academy of Traditional Chinese Medicine, Xi'an, 710003, P. R. China.
Adv Mater. 2025 Feb;37(5):e2413614. doi: 10.1002/adma.202413614. Epub 2024 Dec 17.
Clear-cut evidence has linked defective autophagy to Alzheimer's disease (AD). Recent studies underscore a unique hurdle in AD neuronal autophagy: impaired retrograde axonal transport of autophagosomes, potent enough to induce autophagic stress and neurodegeneration. Nonetheless, pertinent therapy is unavailable. Here, a novel combinational therapy composed of siROCK2 and lithospermic acid B (LA) is introduced, tailored to dredge blocked axonal autophagy by multi-mitigating microtubule disruption, ATP depletion, oxidative stress, and autophagy initiation impediments in AD. Leveraging the recent discovery of multi-interactions between polyphenol LA and siRNA, ε-Poly-L-lysine, and anionic lipid nanovacuoles, LA and siROCK2 are successfully co-loaded into a fresh nano-drug delivery system, LIP@PL-LA/siRC, via a ratio-flexible and straightforward fabrication process. Further modification with the TPL peptide onto LIP@PL-LA/siRC creates a brain-neuron targeted, biocompatible, and pluripotent nanomedicine, named "Nano-dredger" (T-LIP@PL-LA/siRC). Nano-dredger efficiently accelerates axonal retrograde transport and lysosomal degradation of autophagosomes, thereby facilitating the clearance of neurotoxic proteins, improving neuronal complexity, and alleviating memory defects in 3×Tg-AD transgenic mice. This study provides a fresh and flexible polyphenol/siRNA co-delivery paradigm and furnishes conceptual proof that dredging axonal autophagy represents a promising AD therapeutic avenue.
确凿的证据已将自噬缺陷与阿尔茨海默病(AD)联系起来。最近的研究强调了AD神经元自噬中的一个独特障碍:自噬体逆行轴突运输受损,其严重程度足以诱发自噬应激和神经退行性变。然而,目前尚无相关的治疗方法。在此,我们引入了一种由siROCK2和紫草酸B(LA)组成的新型联合疗法,旨在通过多方面减轻AD中的微管破坏、ATP耗竭、氧化应激和自噬起始障碍来疏通受阻的轴突自噬。利用最近发现的多酚LA与siRNA、ε-聚-L-赖氨酸和阴离子脂质纳米空泡之间的多重相互作用,LA和siROCK2通过一种比例灵活且简单的制备工艺成功共载入一种新型纳米药物递送系统LIP@PL-LA/siRC。用TPL肽对LIP@PL-LA/siRC进行进一步修饰,可创建一种靶向脑神经元、具有生物相容性且具有多能性的纳米药物,命名为“纳米疏通剂”(T-LIP@PL-LA/siRC)。纳米疏通剂能有效加速自噬体的轴突逆行运输和溶酶体降解,从而促进神经毒性蛋白的清除,改善神经元复杂性,并减轻3×Tg-AD转基因小鼠的记忆缺陷。本研究提供了一种全新且灵活的多酚/siRNA共递送模式,并提供了概念证明,即疏通轴突自噬是一条有前景的AD治疗途径。