After thirty years of intensive research shaping and optimizing the technology, the approval of the first mRNA-based formulation by the EMA and FDA in order to stop the COVID-19 pandemic was a breakthrough in mRNA research. The astonishing success of these vaccines have brought the mRNA platform into the spotlight of the scientific community. The remarkable persistence of the groundwork is mainly attributed to the exceptional benefits of mRNA application, including the biological origin, immediate but transitory mechanism of action, non-integrative properties, safe and relatively simple manufacturing as well as the flexibility to produce any desired protein. Based on these advantages, a practical implementation of in vitro transcribed mRNA has been considered in most areas of medicine. In this review, we discuss the key preconditions for the rise of the mRNA in the medical field, including the unique structural and functional features of the mRNA molecule and its vehicles, which are crucial aspects for a production of potent mRNA-based therapeutics. Further, we focus on the utility of mRNA tools particularly in the scope of regenerative medicine, i.e. cell reprogramming approaches or manipulation strategies for targeted tissue restoration. Finally, we highlight the strong clinical potential but also the remaining hurdles to overcome for the mRNA-based regenerative therapy, which is only a few steps away from becoming a reality.

经过三十年的深入研究塑造和优化技术，EMA 和 FDA 批准了第一个基于 mRNA 的制剂以阻止 COVID-19 大流行，这是 mRNA 研究的突破。这些疫苗的惊人成功使 mRNA 平台成为科学界关注的焦点。基础工作的显着持久性主要归功于 mRNA 应用的特殊优势，包括生物起源、直接但短暂的作用机制、非整合特性、安全且相对简单的制造以及生产任何所需蛋白质的灵活性。基于这些优点，体外的实际实施大多数医学领域都考虑了转录的 mRNA。在这篇综述中，我们讨论了 mRNA 在医学领域崛起的关键先决条件，包括 mRNA 分子及其载体的独特结构和功能特征，这是生产有效的基于 mRNA 的疗法的关键方面。此外，我们专注于 mRNA 工具的实用性，特别是在再生医学领域，即用于目标组织修复的细胞重编程方法或操作策略。最后，我们强调了基于 mRNA 的再生疗法的强大临床潜力以及需要克服的剩余障碍，这离成为现实只有几步之遥。