Peng Fengli, Qiu Zhimei, Wang Zimu, Li Fuhai, Zhou Fating, Yuan Shuai, Yuan Jinsong, Wang Yan, Zhao Yongchao, Li Chaofu, Shi Bei
Department of Cardiology, Affiliated Hospital of Zunyi Medical University, Zunyi, China.
Department of Cardiology, Zhongshan Hospital, Fudan University, Shanghai Institute of Cardiovascular Diseases, Shanghai, China.
Theranostics. 2025 Jul 2;15(15):7779-7801. doi: 10.7150/thno.111503. eCollection 2025.
mRNA serves as a versatile platform for the expression of paracrine factors, thereby promoting cardioprotection and regeneration. In recent years, mRNA and gene editing technologies have emerged as innovative tools for tackling complex diseases. Among these, mRNA therapeutics offer distinct advantages, including favorable immunological properties, strong safety profiles, and superior flexibility compared to conventional gene-based vaccines. Specifically, they can elicit a balanced immune response-encompassing both cellular and humoral immunity-without being limited by MHC haplotypes. Furthermore, mRNA therapy represents a particularly safe treatment modality. As a minimal and transient carrier of genetic information that does not integrate into the host genome, it significantly reduces the risk of insertional mutagenesis. Importantly, mRNA can be used to express virtually any protein without requiring changes to the production process, thus offering substantial flexibility in drug development. Collectively, these attributes highlight the great potential of mRNA as a next-generation therapeutic platform, particularly for cardiovascular diseases. This review summarizes recent progress in the development and application of mRNA-based drug formulations for cardiovascular therapy.
信使核糖核酸(mRNA)是旁分泌因子表达的通用平台,从而促进心脏保护和再生。近年来,mRNA和基因编辑技术已成为应对复杂疾病的创新工具。其中,mRNA疗法具有独特优势,包括良好的免疫特性、强大的安全性以及与传统基因疫苗相比具有更高的灵活性。具体而言,它们可以引发包括细胞免疫和体液免疫在内的平衡免疫反应,而不受主要组织相容性复合体(MHC)单倍型的限制。此外,mRNA疗法是一种特别安全的治疗方式。作为不整合到宿主基因组中的最小且短暂的遗传信息载体,它显著降低了插入诱变的风险。重要的是,mRNA可用于表达几乎任何蛋白质,而无需改变生产过程,从而在药物开发中提供了极大的灵活性。总体而言,这些特性凸显了mRNA作为下一代治疗平台的巨大潜力,尤其是在心血管疾病治疗方面。本综述总结了用于心血管治疗的基于mRNA的药物制剂开发和应用的最新进展。
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