Telkoparan-Akillilar Pelin, Chichiarelli Silvia, Tucci Paolo, Saso Luciano
Department of Medical Biology, Faculty of Medicine, Gazi University, Ankara, Türkiye.
Department of Biochemical Sciences "A. Rossi-Fanelli", Sapienza University of Rome, Rome, Italy.
Front Cell Dev Biol. 2025 Apr 7;13:1569101. doi: 10.3389/fcell.2025.1569101. eCollection 2025.
MicroRNAs (miRNAs) are small, non-coding RNA molecules that play a pivotal role in the post-transcriptional regulation of gene expression. Over the past decade, they have emerged as key regulators in cancer progression, influencing different cellular processes such as proliferation, apoptosis, metastasis, and immune evasion. Their unique ability to target multiple genes simultaneously makes miRNAs highly attractive as potential therapeutic agents in oncology. However, several challenges have hindered their direct clinical application, most notably their inherent instability in biological fluids, rapid degradation by nucleases, and inefficient delivery to specific tumor sites. Additionally, off-target effects and the potential for toxicity further complicate the therapeutic use of miRNAs. Nanomedicine offers a promising solution to these challenges by enabling the development of advanced platforms for the stable, safe, and targeted delivery of miRNAs. Nanoparticle-based delivery systems, such as liposomes, polymeric nanoparticles, and inorganic nanocarriers, can protect miRNAs from degradation, improve their bioavailability, and allow for precise tumor targeting through passive or active targeting mechanisms. These nanocarriers can also be engineered to release miRNAs in response to specific stimuli within the tumor microenvironment, enhancing therapeutic efficacy while minimizing side effects. This review will explore the integration of miRNAs with nanotechnology, focusing on various nanoparticle formulations and their roles in enhancing miRNA stability, specificity, and function in cancer treatment. In addition, we will discuss current advances in preclinical and clinical applications, highlight promising tumor-targeting strategies, and address the remaining challenges such as toxicity, immunogenicity, and scalability. Future research should focus on overcoming these barriers, ultimately paving the way for the widespread adoption of personalized miRNA-based nanomedicine in cancer therapy.
微小RNA(miRNA)是一类小的非编码RNA分子,在基因表达的转录后调控中起关键作用。在过去十年中,它们已成为癌症进展的关键调节因子,影响着增殖、凋亡、转移和免疫逃逸等不同细胞过程。miRNA能够同时靶向多个基因的独特能力使其作为肿瘤学中的潜在治疗药物极具吸引力。然而,一些挑战阻碍了它们的直接临床应用,最显著的是它们在生物流体中固有的不稳定性、被核酸酶快速降解以及向特定肿瘤部位的递送效率低下。此外,脱靶效应和潜在的毒性使miRNA的治疗应用更加复杂。纳米医学通过开发用于稳定、安全和靶向递送miRNA的先进平台,为这些挑战提供了一个有前景的解决方案。基于纳米颗粒的递送系统,如脂质体、聚合物纳米颗粒和无机纳米载体,可以保护miRNA不被降解,提高其生物利用度,并通过被动或主动靶向机制实现精确的肿瘤靶向。这些纳米载体还可以被设计成在肿瘤微环境中响应特定刺激释放miRNA,提高治疗效果同时最小化副作用。本综述将探讨miRNA与纳米技术的整合,重点关注各种纳米颗粒制剂及其在增强miRNA在癌症治疗中的稳定性、特异性和功能方面的作用。此外,我们将讨论临床前和临床应用的当前进展,突出有前景的肿瘤靶向策略,并解决毒性、免疫原性和可扩展性等 remaining challenges(原文此处有误,疑为remaining challenges,可译为“剩余挑战”)。未来的研究应专注于克服这些障碍,最终为基于个性化miRNA的纳米医学在癌症治疗中的广泛应用铺平道路。