RNA免疫疗法：革新癌症和自身免疫性疾病的治疗方法。

RNA immunotherapy: revolutionizing cancer and autoimmune disease treatments.

作者信息

Savguira Margarita, Chen David X W, Dong Songtao, Li Bowen

机构信息

Institute of Biomedical Engineering, University of Toronto, Toronto, ON M5S 3G9, Canada.

Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, ON M5S 3M2, Canada.

出版信息

Trends Mol Med. 2025 Jul 12. doi: 10.1016/j.molmed.2025.06.008.

DOI:10.1016/j.molmed.2025.06.008

PMID:40653413

Abstract

RNA immunotherapy offers a versatile and scalable platform for reprogramming immune responses in cancer and autoimmunity. Advances in linear mRNA, self-amplifying RNA (saRNA), and circular RNA (circRNA) enable the programmable delivery of therapeutic proteins with improved stability, expression, and targeting. In cancer, RNA platforms have shown clinical promise in neoantigen vaccines, cytokine therapies, and chimeric antigen receptor (CAR) immune cell engineering. In autoimmunity, RNA strategies are being developed to induce antigen-specific tolerance, expand regulatory T cells (Tregs), and deliver anti-inflammatory mediators. Structural optimizations and targeted delivery systems further enhance therapeutic efficacy and safety. This review discusses recent clinical progress, mechanistic foundations, and emerging directions for RNA immunotherapy as a broadly applicable platform across cancer and autoimmune disease contexts.

摘要

RNA免疫疗法为在癌症和自身免疫中重新编程免疫反应提供了一个通用且可扩展的平台。线性mRNA、自我扩增RNA（saRNA）和环状RNA（circRNA）的进展使得治疗性蛋白质能够以可编程的方式递送，稳定性、表达和靶向性都得到了改善。在癌症方面，RNA平台在新抗原疫苗、细胞因子疗法和嵌合抗原受体（CAR）免疫细胞工程中已展现出临床前景。在自身免疫中，正在开发RNA策略以诱导抗原特异性耐受、扩增调节性T细胞（Tregs）并递送抗炎介质。结构优化和靶向递送系统进一步提高了治疗效果和安全性。本综述讨论了RNA免疫疗法作为一个广泛适用于癌症和自身免疫性疾病的平台的近期临床进展、机制基础和新兴方向。