Lipid nanoparticles for mRNA delivery.
Drug delivery
Drug development
Journal
Nature reviews. Materials
ISSN: 2058-8437
Titre abrégé: Nat Rev Mater
Pays: England
ID NLM: 101692128
Informations de publication
Date de publication:
2021
2021
Historique:
accepted:
19
07
2021
pubmed:
17
8
2021
medline:
17
8
2021
entrez:
16
8
2021
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 34394960
doi: 10.1038/s41578-021-00358-0
pii: 358
pmc: PMC8353930
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
1078-1094Subventions
Organisme : NIBIB NIH HHS
ID : R01 EB000244
Pays : United States
Organisme : NIGMS NIH HHS
ID : R35 GM119679
Pays : United States
Organisme : NIBIB NIH HHS
ID : R37 EB000244
Pays : United States
Informations de copyright
© Springer Nature Limited 2021, corrected publication 2021.
Déclaration de conflit d'intérêts
Competing interestsY.D. is a scientific advisory board member of Oncorus, Inc. and serves as a consultant of Rubius Therapeutics. T.Z. is an employee of Moderna, Inc. R.L. is a founding scientific advisory board member of Alnylam and a founder and board member of Moderna, Inc. A list of entities with which R.L. is involved, compensated or uncompensated is provided in the supplementary information. X.H. declares no competing interests.
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