小干扰RNA（siRNA）在癌症治疗的临床应用方面的进展为精准医学开辟了新的可能性。作为强大的遗传工具，siRNA已经显示出针对和抑制与癌症进展相关的特定基因表达的潜力。通过将siRNA纳入纳米颗粒中，其效果进一步增强，纳米颗粒可以保护siRNA免受降解，并实现靶向递送。然而，尽管这些有希望的发展，siRNA基于癌症治疗的临床转化仍然存在一些挑战。该综述回顾了siRNA在癌症治疗中的临床应用的进展和挑战。该综述突出了使用siRNA载荷纳米颗粒作为一种有效的递送系统，用于优化各种类型的癌症中siRNA的功效，并强调了siRNA基于治疗作为一种遗传方法来克服与常规化疗药物相关的严重毒性和器官损伤等限制的潜力。此外，它强调了一些关键的挑战，包括非特异性效应、血清中siRNA的酶降解、肿瘤定位率低、稳定性问题和循环快速清除等，这些挑战需要解决以实现siRNA基于癌症治疗的成功临床开发。尽管存在这些挑战，该综述确定了从实验室到临床环境推进siRNA技术的重要途径。siRNA纳载纳米颗粒用于癌症治疗的持续进展显示出潜在的抗肿瘤活性和安全性。通过了解当前的siRNA基于治疗的现状并解决现有的挑战，我们旨在为将siRNA技术作为改进的癌症患者治疗选择有效地应用于临床设置铺平道路。© 2023. 作者，由Springer Nature B.V.独家授权。

Advancements in the clinical applications of small interfering RNA (siRNA) in cancer therapy have opened up new possibilities for precision medicine. siRNAs, as powerful genetic tools, have shown potential in targeting and suppressing the expression of specific genes associated with cancer progression. Their effectiveness has been further enhanced by incorporating them into nanoparticles, which protect siRNAs from degradation and enable targeted delivery. However, despite these promising developments, several challenges persist in the clinical translation of siRNA-based cancer therapy. This comprehensive review explores the progress and challenges associated with the clinical applications of siRNA in cancer therapy. This review highlights the use of siRNA-loaded nanoparticles as an effective delivery system for optimizing siRNA efficacy in various types of carcinomas and the potential of siRNA-based therapy as a genetic approach to overcome limitations associated with conventional chemotherapeutic agents, including severe drug toxicities and organ damage. Moreover, it emphasizes on the key challenges, including off-target effects, enzymatic degradation of siRNAs in serum, low tumor localization, stability issues, and rapid clearance from circulation that need to be addressed for successful clinical development of siRNA-based cancer therapy. Despite these challenges, the review identifies significant avenues for advancing siRNA technology from the laboratory to clinical settings. The ongoing progress in siRNA-loaded nanoparticles for cancer treatment demonstrates potential antitumor activities and safety profiles. By understanding the current state of siRNA-based therapy and addressing the existing challenges, we aim to pave the way for translating siRNA technology into effective oncologic clinics as an improved treatment options for cancer patients.© 2023. The Author(s), under exclusive licence to Springer Nature B.V.