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Lipid nanoparticles for mRNA delivery
Messenger RNA (mRNA) has emerged as a new category of therapeutic agent to prevent and treat various diseases. 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. Lipid nanopart...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8353930/ https://www.ncbi.nlm.nih.gov/pubmed/34394960 http://dx.doi.org/10.1038/s41578-021-00358-0 |
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author | Hou, Xucheng Zaks, Tal Langer, Robert Dong, Yizhou |
author_facet | Hou, Xucheng Zaks, Tal Langer, Robert Dong, Yizhou |
author_sort | Hou, Xucheng |
collection | PubMed |
description | Messenger RNA (mRNA) has emerged as a new category of therapeutic agent to prevent and treat various diseases. 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. 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. 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. 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. Finally, we give an outlook to future possibilities and remaining challenges for this promising technology. |
format | Online Article Text |
id | pubmed-8353930 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Nature Publishing Group UK |
record_format | MEDLINE/PubMed |
spelling | pubmed-83539302021-08-10 Lipid nanoparticles for mRNA delivery Hou, Xucheng Zaks, Tal Langer, Robert Dong, Yizhou Nat Rev Mater Review Article Messenger RNA (mRNA) has emerged as a new category of therapeutic agent to prevent and treat various diseases. 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. 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. 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. 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. Finally, we give an outlook to future possibilities and remaining challenges for this promising technology. Nature Publishing Group UK 2021-08-10 2021 /pmc/articles/PMC8353930/ /pubmed/34394960 http://dx.doi.org/10.1038/s41578-021-00358-0 Text en © Springer Nature Limited 2021, corrected publication 2021 This article is made available via the PMC Open Access Subset for unrestricted research re-use and secondary analysis in any form or by any means with acknowledgement of the original source. These permissions are granted for the duration of the World Health Organization (WHO) declaration of COVID-19 as a global pandemic. |
spellingShingle | Review Article Hou, Xucheng Zaks, Tal Langer, Robert Dong, Yizhou Lipid nanoparticles for mRNA delivery |
title | Lipid nanoparticles for mRNA delivery |
title_full | Lipid nanoparticles for mRNA delivery |
title_fullStr | Lipid nanoparticles for mRNA delivery |
title_full_unstemmed | Lipid nanoparticles for mRNA delivery |
title_short | Lipid nanoparticles for mRNA delivery |
title_sort | lipid nanoparticles for mrna delivery |
topic | Review Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8353930/ https://www.ncbi.nlm.nih.gov/pubmed/34394960 http://dx.doi.org/10.1038/s41578-021-00358-0 |
work_keys_str_mv | AT houxucheng lipidnanoparticlesformrnadelivery AT zakstal lipidnanoparticlesformrnadelivery AT langerrobert lipidnanoparticlesformrnadelivery AT dongyizhou lipidnanoparticlesformrnadelivery |