Notably, two authorized coronavirus disease 2019 (COVID-19) vaccines, mRNA-1273 (refs 19, 20) and BNT162b 21, use lipid nanoparticles to deliver antigen mRNA. In particular, lipid nanoparticles have been thoroughly investigated and successfully entered the clinic for the delivery of small molecules 18, siRNA drugs 18 and mRNA 19, 20, 21. However, targeted delivery and endosomal escape remain challenging for mRNA delivery systems, highlighting the need for safe and effective mRNA delivery materials.Ī variety of materials have been developed for mRNA delivery, including lipids, lipid-like materials, polymers and protein derivatives 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17. To achieve therapeutic effects, mRNA molecules have to reach specific target cells and produce sufficient proteins of interest.
MRNA has shown therapeutic potential in a range of applications, including viral vaccines, protein replacement therapies, cancer immunotherapies, cellular reprogramming and genome editing 2, 4, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17. Finally, we give an outlook to future possibilities and remaining challenges for this promising technology. We then consider key points for the clinical translation of lipid nanoparticle–mRNA formulations, including good manufacturing practice, stability, storage and safety, and highlight preclinical and clinical studies of lipid nanoparticle–mRNA therapeutics for infectious diseases, cancer and genetic disorders. In this Review, we discuss the design of lipid nanoparticles for mRNA delivery and examine physiological barriers and possible administration routes for lipid nanoparticle–mRNA systems. Lipid nanoparticles have successfully entered the clinic for the delivery of mRNA in particular, lipid nanoparticle–mRNA vaccines are now in clinical use against coronavirus disease 2019 (COVID-19), which marks a milestone for mRNA therapeutics. To function in vivo, mRNA requires safe, effective and stable delivery systems that protect the nucleic acid from degradation and that allow cellular uptake and mRNA release.
Messenger RNA (mRNA) has emerged as a new category of therapeutic agent to prevent and treat various diseases.
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