Recent Advances in Site-Specific Lipid Nanoparticles for mRNA Delivery

IF 4.8 Q2 NANOSCIENCE & NANOTECHNOLOGY

ACS Nanoscience Au Pub Date : 2023-03-30 DOI:10.1021/acsnanoscienceau.2c00062

Xiao Xu, and , Tian Xia*,

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引用次数: 2

Abstract

The success of mRNA vaccines during the COVID-19 pandemic has greatly accelerated the development of mRNA therapy. mRNA is a negatively charged nucleic acid that serves as a template for protein synthesis in the ribosome. Despite its utility, the instability of mRNA requires suitable carriers for in vivo delivery. Lipid nanoparticles (LNPs) are employed to protect mRNA from degradation and enhance its intracellular delivery. To further optimize the therapeutic efficacy of mRNA, site-specific LNPs have been developed. Through local or systemic administration, these site-specific LNPs can accumulate in specific organs, tissues, or cells, allowing for the intracellular delivery of mRNA to specific cells and enabling the exertion of local or systemic therapeutic effects. This not only improves the efficiency of mRNA therapy but also reduces off-target adverse effects. In this review, we summarize recent site-specific mRNA delivery strategies, including different organ- or tissue-specific LNP after local injection, and organ-specific or cell-specific LNP after intravenous injection. We also provide an outlook on the prospects of mRNA therapy.

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用于mRNA递送的位点特异性脂质纳米颗粒的最新进展

mRNA疫苗在新冠肺炎大流行期间的成功极大地加速了mRNA疗法的发展。信使核糖核酸是一种带负电荷的核酸，在核糖体中作为蛋白质合成的模板。尽管信使核糖核酸具有实用性，但其不稳定性需要合适的载体进行体内递送。脂质纳米颗粒（LNPs）用于保护信使核糖核酸免受降解并增强其细胞内递送。为了进一步优化信使核糖核酸的治疗效果，已经开发了位点特异性LNP。通过局部或全身给药，这些位点特异性LNP可以在特定器官、组织或细胞中积累，从而允许mRNA在细胞内递送到特定细胞，并能够发挥局部或全身治疗效果。这不仅提高了信使核糖核酸治疗的效率，而且减少了脱靶不良反应。在这篇综述中，我们总结了最近的位点特异性信使核糖核酸递送策略，包括局部注射后的不同器官或组织特异性LNP，以及静脉注射后的器官特异性或细胞特异性LNP。我们还对信使核糖核酸治疗的前景进行了展望。

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来源期刊

ACS Nanoscience Au 材料科学、纳米科学-

CiteScore

4.20

自引率

0.00%

发文量

期刊介绍： ACS Nanoscience Au is an open access journal that publishes original fundamental and applied research on nanoscience and nanotechnology research at the interfaces of chemistry biology medicine materials science physics and engineering.The journal publishes short letters comprehensive articles reviews and perspectives on all aspects of nanoscience and nanotechnology:synthesis assembly characterization theory modeling and simulation of nanostructures nanomaterials and nanoscale devicesdesign fabrication and applications of organic inorganic polymer hybrid and biological nanostructuresexperimental and theoretical studies of nanoscale chemical physical and biological phenomenamethods and tools for nanoscience and nanotechnologyself- and directed-assemblyzero- one- and two-dimensional materialsnanostructures and nano-engineered devices with advanced performancenanobiotechnologynanomedicine and nanotoxicologyACS Nanoscience Au also publishes original experimental and theoretical research of an applied nature that integrates knowledge in the areas of materials engineering physics bioscience and chemistry into important applications of nanomaterials.