mRNA-based gene therapy has remarkable therapeutic potential in various disease area including infectious disease, cancer and rare disease. The success of mRNA-based gene therapies highly replies on the functional delivery of mRNA into the cytoplasm of targeted cells. However, due to the instability and inefficient <i>in vivo</i> delivery of mRNA, its application is heavily restricted. Recent advances in lipid nanoparticles (LNP) have profoundly driven the implementation of mRNA-based gene therapies in clinical settings, which attribute to the design and optimization of lipid component that enables safe and efficient delivery of mRNA. In this review, we first discuss and highlight the main factors impeding the successful target cell transfection and tropism of lipid nanoparticle-coated mRNA <i>in vivo</i>. In addition, the evolutionary design, characterization and optimization of lipid library for successful LNP development through the past decades will be summarized, with particular interest in recent progress in the development of LNP mediated mRNA-based gene therapies and their clinical utility in various disease treatment.

Advances in lipid nanoparticles for mRNA delivery: From concept to clinical intervention

ACS Fall 2021

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