Khan Mudassir, Musazzi Umberto M, Manellari Sara, Mouawad Nayla, Damiano Giulia, Rinaldi Raffaella, Raucci Angela, Costantino Sarah, Paneni Francesco, Minghetti Paola, Pompilio Giulio, Vinci Maria Cristina
Department of Biomedical, Surgical and Dental Sciences, University of Milan, Milan 20100, Italy; Centro Cardiologico Monzino IRCCS, Via C. Parea 4, Milan 20138, Italy.
Department of Pharmaceutical Sciences, University of Milan, 20133, Italy.
Pharmacol Res. 2025 Aug;218:107847. doi: 10.1016/j.phrs.2025.107847. Epub 2025 Jul 1.
Messenger RNA (mRNA) is emerging as a new tool for therapeutic intervention, providing a transient, programmable platform for the treatment of complex diseases. Unlike DNA-based therapies, mRNA does not integrate into the genome and is considered more safe. The success of mRNA vaccines, combined with advances in mRNA technology and unmet needs in cardiovascular disease (CVD), has led to increased interest in expanding the use of this technology beyond infectious diseases. However, there are several challenges to overcome, including mRNA stability, delivery efficiency, immunogenicity, and regulatory standardization. This review examines advances in mRNA design, delivery platforms, and therapeutic applications in CVDs, emphasizing the need for a clear regulatory framework to facilitate clinical translation. The establishment of consistent regulatory guidelines will be a critical step in ensuring the safety and efficacy of mRNA-based cardiovascular therapies, potentially facilitating their widespread adoption. The future direction of research in mRNA formulation, personalized medicine approaches, and the refinement of regulatory policies will determine the landscape of next-generation cardiovascular therapeutics.
信使核糖核酸（mRNA）正成为一种用于治疗干预的新工具，为复杂疾病的治疗提供了一个短暂的、可编程的平台。与基于DNA的疗法不同，mRNA不会整合到基因组中，被认为更安全。mRNA疫苗的成功，再加上mRNA技术的进步以及心血管疾病（CVD）未满足的需求，使得人们对将该技术的应用扩展到传染病之外的领域兴趣大增。然而，仍有几个挑战需要克服，包括mRNA稳定性、递送效率、免疫原性和监管标准化。本综述探讨了mRNA设计、递送平台以及在心血管疾病中的治疗应用方面的进展，强调需要一个明确的监管框架以促进临床转化。建立一致的监管指南将是确保基于mRNA的心血管疗法的安全性和有效性、潜在促进其广泛应用的关键一步。mRNA制剂、个性化医疗方法以及监管政策完善方面的未来研究方向将决定下一代心血管治疗的格局。
