RNA信使分子（mRNA）近期成为治疗和预防多种疾病的有吸引力的替代选择，范围从癌症和阿尔茨海默病到COVID-19，具有显着的临床效果。体外转录的mRNA已经被工程成类似天然mRNA的结构，用于疫苗、肿瘤免疫疗法和蛋白质替代疗法。在过去几十年中，揭示mRNA的分子途径，控制其可翻译性和稳定性，以及其进化防御机制取得了显著进展。然而，许多未解决的结构、生物和技术困难阻碍了mRNA系统传递以实现更安全的人体摄入。设计和制造mRNA以及选择创新的递送载体的进展是解决未解决的问题并实现mRNA药物的全部潜力所必需的。尽管在改善mRNA药物的细胞内递送方面已经做出了实质性的努力，在不同途径的不同应用方面仍存在挑战。本研究考察了mRNA治疗的当前进展、设计生物材料和递送策略的进展、临床可追溯性的现有翻译难题以及克服与mRNA相关的任何挑战的前景。 ©2022年。韩国药学学会。

Messenger RNA (mRNA) recently emerged as an appealing alternative to treat and prevent diseases ranging from cancer and Alzheimer's disease to COVID-19 with significant clinical outputs. The in vitro-transcribed mRNA has been engineered to mimic the structure of natural mRNA for vaccination, cancer immunotherapy and protein replacement therapy. In past decades, significant progress has been noticed in unveiling the molecular pathways of mRNA, controlling its translatability and stability, and its evolutionary defense mechanism. However, numerous unsolved structural, biological, and technical difficulties hamper the successful implementation of systemic delivery of mRNA for safer human consumption. Advances in designing and manufacturing mRNA and selecting innovative delivery vehicles are mandatory to address the unresolved issues and achieve the full potential of mRNA drugs. Despite the substantial efforts made to improve the intracellular delivery of mRNA drugs, challenges associated with diverse applications in different routes still exist. This study examines the current progress of mRNA therapeutics and advancements in designing biomaterials and delivery strategies, the existing translational challenges of clinical tractability and the prospects of overcoming any challenges related to mRNA.© 2022. The Pharmaceutical Society of Korea.