mRNA therapeutics: Transforming medicine through innovation in design, delivery, and disease treatment

作者：Mark Akinola Ige, Xiaohu Ren, Yang Yang, Haiping Zhang, Chenguang Shen, Yushan Jiang, Junxin Li, Xiaochun Wan · 发表于：Molecular Therapy — Nucleic Acids · 年份：2025 · DOI：10.1016/j.omtn.2025.102721 · 被引用次数：19 · 研究领域：RNA Interference and Gene Delivery、Virus-based gene therapy research、Cancer Research and Treatments

mRNA therapeutics have revolutionized medicine, offering a versatile platform to address previously untreatable diseases. Recent advancements in biotechnology have enabled the efficient production of functional proteins, antibodies, and peptides via mRNA, providing rapid and adaptable solutions for vaccine development and therapeutic interventions. The success of mRNA vaccines, exemplified during the COVID-19 pandemic, underscores their potential for combating infectious diseases with unparalleled speed and scalability. This review explores the latest developments in mRNA technology, including innovations in design, delivery, and disease treatment; modulation of immune response; and the role of AI. Particular emphasis is placed on optimizing mRNA constructs to maximize therapeutic efficacy, new delivery vehicles for mRNA, and the modulations of immune response evoked by mRNA vaccines. The potential applications of mRNA therapeutics in genetic disorders, infectious diseases, and cancer are highlighted, alongside a discussion of existing challenges such as delivery efficiency and production scalability. By integrating molecular biology, RNA technology, and nanotechnology, mRNA therapeutics hold the promise of transforming precision medicine. These advancements offer hope for patients with complex or intractable conditions, paving the way for a new era in targeted therapies and personalized healthcare.