Barui Ayan K, Amirrad Farideh, Goel Vansh, Ohadi Sharareh, Pala Rajasekharreddy, Mohieldin Ashraf M, Nauli Andromeda M, Nauli Surya M
Department of Biomedical and Pharmaceutical Sciences, Chapman University, Irvine, CA, 92618, USA.
Department of Pharmaceutical Sciences, School of Pharmacy, University of California Irvine, Irvine, CA, 92697, USA.
Bioact Mater. 2025 May 8;51:70-82. doi: 10.1016/j.bioactmat.2025.04.015. eCollection 2025 Sep.
Primary cilia are hair-like structured cell organelles functioning as cellular antennae with chemosensory and mechanosensory roles. Dysfunction of cilia results in ciliopathies, including renovascular diseases (polycystic kidneys, hypertension, aneurysm), mental impedances (Alzheimer's disease, Parkinson's disease, dementia), metabolic diseases (obesity, developmental skeleton defects), blindness, and so on. With a diameter of 150 nm, cilia can be cleaved, released and circulated in the body as injury biomarkers. We here assessed the potential use of isolated cilia as a novel delivery vehicle. Cilia were isolated from renal epithelial cells, and a specific-targeting chemotherapeutic was designed on the cilia. The data show a remarkable property of cilia to significantly inhibit disease progression with reduced toxicity. These findings emphasize the prospective biomedical applications of primary cilia in generating novel drug, gene, protein, and other delivery systems to treat diverse pathological conditions. While there are many synthetic drug delivery vehicles, our studies demonstrate for the first time that physiologically produced primary cilia can be adapted as a unique carrier system.
原发性纤毛是毛发状结构的细胞器,作为具有化学感应和机械感应作用的细胞触角发挥功能。纤毛功能障碍会导致纤毛病,包括肾血管疾病(多囊肾、高血压、动脉瘤)、智力障碍(阿尔茨海默病、帕金森病、痴呆症)、代谢疾病(肥胖、发育性骨骼缺陷)、失明等等。纤毛直径为150纳米,可作为损伤生物标志物在体内裂解、释放并循环。我们在此评估了分离出的纤毛作为新型递送载体的潜在用途。从肾上皮细胞中分离出纤毛,并在纤毛上设计了一种特异性靶向化疗药物。数据显示纤毛具有显著特性,能显著抑制疾病进展且毒性降低。这些发现强调了原发性纤毛在开发新型药物、基因、蛋白质及其他递送系统以治疗多种病理状况方面的潜在生物医学应用。虽然有许多合成药物递送载体,但我们的研究首次证明生理产生的原发性纤毛可被用作独特的载体系统。