PDF(Pubmed)
Abstract：
The idea of mRNA therapy had been conceived for decades before it came into reality during the Covid-19 pandemic. The mRNA vaccine emerges as a powerful and general tool against new viral infections, largely due to its versatility and rapid development. In addition to prophylactic vaccines, mRNA technology also offers great promise for new applications as a versatile drug modality. However, realizing the conceptual potential faces considerable challenges, such as minimal immune stimulation, high and long-term expression, and efficient delivery to target cells and tissues. Here we review the applications of mRNA-based therapeutics, with emphasis on the innovative design and future challenges/solutions. In addition, we also discuss the next generation of mRNA therapy, including circular mRNA and self-amplifying RNAs. We aim to provide a conceptual overview and outlook on mRNA therapeutics beyond prophylactic vaccines.
摘要：
mRNA治疗的想法在新冠肺炎大流行期间成为现实之前已经构思了几十年。mRNA疫苗成为对抗新的病毒感染的强大而通用的工具，很大程度上是由于其多功能性和快速发展。除了预防性疫苗，mRNA技术也为作为通用药物形式的新应用提供了巨大的希望。然而,实现概念潜力面临相当大的挑战，比如最小的免疫刺激，高和长期表达，并有效递送到靶细胞和组织。在这里，我们回顾了基于mRNA的疗法的应用，强调创新设计和未来的挑战/解决方案。此外,我们还讨论了下一代mRNA治疗，包括环状mRNA和自扩增RNA。我们旨在提供预防性疫苗以外的mRNA治疗的概念概述和展望。
