基于脂质纳米粒的 mRNA 治疗的开发和应用。
Development and applications of mRNA treatment based on lipid nanoparticles.
机构信息
Key Laboratory of Biotechnology and Bioresources Utilization of Ministry of Education, Dalian Minzu University, Dalian 116600, China.
Centenary Institute, The University of Sydney, Sydney, NSW 2050, Australia.
出版信息
Biotechnol Adv. 2023 Jul-Aug;65:108130. doi: 10.1016/j.biotechadv.2023.108130. Epub 2023 Mar 17.
Nucleic acid-based therapies such as messenger RNA have the potential to revolutionize modern medicine and enhance the performance of existing pharmaceuticals. The key challenges of mRNA-based therapies are delivering the mRNA safely and effectively to the target tissues and cells and controlling its release from the delivery vehicle. Lipid nanoparticles (LNPs) have been widely studied as drug carriers and are considered to be state-of-the-art technology for nucleic acid delivery. In this review, we begin by presenting the advantages and mechanisms of action of mRNA therapeutics. Then we discuss the design of LNP platforms based on ionizable lipids and the applications of mRNA-LNP vaccines for prevention of infectious diseases and for treatment of cancer and various genetic diseases. Finally, we describe the challenges and future prospects of mRNA-LNP therapeutics.
核酸类疗法，如信使 RNA，具有改变现代医学和提高现有药物性能的潜力。基于 mRNA 的疗法的关键挑战是安全有效地将 mRNA 递送到靶组织和细胞，并控制其从递药载体中的释放。脂质纳米粒（LNPs）已被广泛研究作为药物载体，被认为是核酸递药的最先进技术。在这篇综述中，我们首先介绍了 mRNA 治疗的优点和作用机制。然后我们讨论了基于可离子化脂质的 LNP 平台的设计以及 mRNA-LNP 疫苗在预防传染病以及治疗癌症和各种遗传疾病方面的应用。最后，我们描述了 mRNA-LNP 治疗的挑战和未来前景。
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