Recent advances in lipid nanoparticles for delivery of nucleic acid,mRNA, and gene editing-based therapeutics |
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| Institution: | 1. Department of Chemical Engineering, Carnegie Mellon University, Pittsburgh, PA 15213, United States of America;2. Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, United States of America;3. Department of Biochemistry, Simmons Comprehensive Cancer Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, United States of America;1. Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA;2. Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA;3. Department of Medicine, Division of Pulmonary, Allergy, and Critical Care Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA;4. Department of Bioengineering, University of Pennsylvania, Philadelphia, PA, USA;5. Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA;6. Institute for Immunology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA;7. Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA;8. Institute for Regenerative Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA;9. Genevant Sciences Corporation, Vancouver, BC V5T 4T5, Canada |
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| Abstract: | Lipid nanoparticles (LNPs) are becoming popular as a means of delivering therapeutics, including those based on nucleic acids and mRNA. The mRNA-based coronavirus disease 2019 vaccines are perfect examples to highlight the role played by drug delivery systems in advancing human health. The fundamentals of LNPs for the delivery of nucleic acid- and mRNA-based therapeutics, are well established. Thus, future research on LNPs will focus on addressing the following: expanding the scope of drug delivery to different constituents of the human body, expanding the number of diseases that can be targeted, and studying the change in the pharmacokinetics of LNPs under physiological and pathological conditions. This review article provides an overview of recent advances aimed at expanding the application of LNPs, focusing on the pharmacokinetics and advantages of LNPs. In addition, analytical techniques, library construction and screening, rational design, active targeting, and applicability to gene editing therapy have also been discussed. |
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| Keywords: | Lipid nanoparticle Nucleic acid mRNA Gene editing Base editing Ionizable lipid Targeting DNA barcode Recombination Positron emission tomography |
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