Australian Institute for Bioengineering and Nanotechnology, the University of Queensland, Brisbane, QLD 4072, Australia.
Division of Pharmacoengineering and Molecular Pharmaceutics, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Adv Drug Deliv Rev. 2023 Sep;200:115042. doi: 10.1016/j.addr.2023.115042. Epub 2023 Aug 2.
The concept of using mRNA to produce its own medicine in situ in the body makes it an ideal drug candidate, holding great potential to revolutionize the way we approach medicine. The unique characteristics of mRNA, as well as its customizable biomedical functions, call for the rational design of delivery systems to protect and transport mRNA molecules. In this review, a nanoparticle toolkit is presented for the development of mRNA-based therapeutics from a drug delivery perspective. Nano-delivery systems derived from either natural systems or chemical synthesis, in the nature of organic or inorganic materials, are summarised. Delivery strategies in controlling the tissue targeting and mRNA release, as well as the role of nanoparticles in building and boosting the activity of mRNA drugs, have also been introduced. In the end, our insights into the clinical and translational development of mRNA nano-drugs are presented.
利用 mRNA 在体内原位产生自身药物的概念使其成为一种理想的药物候选物，有望彻底改变我们对待医学的方式。mRNA 的独特特性及其可定制的生物医学功能要求对其进行合理的递送系统设计，以保护和输送 mRNA 分子。在这篇综述中，从药物递送的角度出发，提出了一个基于纳米颗粒的工具包，用于开发基于 mRNA 的治疗方法。总结了源自天然系统或化学合成的纳米递药系统，包括有机或无机材料。还介绍了控制组织靶向和 mRNA 释放的递药策略，以及纳米颗粒在构建和增强 mRNA 药物活性中的作用。最后，提出了我们对 mRNA 纳米药物临床和转化发展的见解。
