本文由小咖机器人翻译整理

期刊来源：Lancet

原文链接：https://doi.org/10.1016/S0140-6736(23)02444-3

摘要内容如下：

mRNA技术及其递送的最新进展使基于mRNA的疗法进入了医学的新时代。mRNA编码的蛋白质具有快速、有效和短暂的性质，无需进入细胞核或基因组整合的风险，这使得它们成为治疗一系列疾病（从传染病到癌症和单基因疾病）的理想工具。基于mRNA的疗法的快速步伐和大规模制造的便利性支持了对COVID-19大流行的全球响应。尽管如此，在mRNA稳定性、表达持续时间、递送效率和靶向性方面仍然存在挑战，以扩大Covid-19疫苗之外的mRNA疗法的适用性。通过从快速扩展的临床前和临床研究中学习，我们可以优化mRNA平台，以满足每种疾病的临床需求。在这里，我们将总结mRNA技术的最新进展。其在疫苗、免疫疗法、蛋白质替代疗法和基因组编辑中的用途；以及将其递送至所需的特定细胞类型和器官，以开发新一代基于靶向mRNA的治疗剂。

英文原文如下：

Abstracts

Recent advances in mRNA technology and its delivery have enabled mRNA-based therapeutics to enter a new era in medicine. The rapid, potent, and transient nature of mRNA-encoded proteins, without the need to enter the nucleus or the risk of genomic integration, makes them desirable tools for treatment of a range of diseases, from infectious diseases to cancer and monogenic disorders. The rapid pace and ease of mass-scale manufacturability of mRNA-based therapeutics supported the global response to the COVID-19 pandemic. Nonetheless, challenges remain with regards to mRNA stability, duration of expression, delivery efficiency, and targetability, to broaden the applicability of mRNA therapeutics beyond COVID-19 vaccines. By learning from the rapidly expanding preclinical and clinical studies, we can optimise the mRNA platform to meet the clinical needs of each disease. Here, we will summarise the recent advances in mRNA technology; its use in vaccines, immunotherapeutics, protein replacement therapy, and genomic editing; and its delivery to desired specific cell types and organs for development of a new generation of targeted mRNA-based therapeutics.

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