实现基于 mRNA 的药物的巨大临床潜力需要持续开发方法，以安全高效地递送生物活性剂且不会引发副作用。在这方面，脂质纳米颗粒已成功用于改善 mRNA 递送并保护货物免受细胞外降解。脂质纳米颗粒的封装是 mRNA 疫苗成功临床应用的一个重要因素，这最终证明了该技术生产批准药物的潜力。在这篇综述中，我们首先描述了 mRNA 修饰、新型脂质的设计以及基于 mRNA 的药物的脂质纳米颗粒成分的开发的当前进展。然后，我们总结了有关 mRNA 疗法的临床前和临床开发的要点。最后，我们讨论与靶向递送系统相关的主题，包括内体逃逸和靶向免疫细胞、肿瘤和器官，用于 mRNA 疫苗和人类疾病的新治疗方式。© 2024。作者。

Realizing the immense clinical potential of mRNA-based drugs will require continued development of methods to safely deliver the bioactive agents with high efficiency and without triggering side effects. In this regard, lipid nanoparticles have been successfully utilized to improve mRNA delivery and protect the cargo from extracellular degradation. Encapsulation in lipid nanoparticles was an essential factor in the successful clinical application of mRNA vaccines, which conclusively demonstrated the technology's potential to yield approved medicines. In this review, we begin by describing current advances in mRNA modifications, design of novel lipids and development of lipid nanoparticle components for mRNA-based drugs. Then, we summarize key points pertaining to preclinical and clinical development of mRNA therapeutics. Finally, we cover topics related to targeted delivery systems, including endosomal escape and targeting of immune cells, tumors and organs for use with mRNA vaccines and new treatment modalities for human diseases.© 2024. The Author(s).