Cited by 130RNA Interference and Gene DeliveryNanoparticle-Based Drug DeliveryDendrimers and Hyperbranched Polymers

Nanoplatforms for mRNA Therapeutics

Meng, Chaoyang, Chen, Zhe, Li, Gang, Welte, Thomas, Shen, Haifa·2021·Advanced Therapeutics

Abstract

mRNA‐based therapeutics are a unique class of drugs for treatment of human diseases. Since in vitro transcribed mRNA molecules have relatively low stability and their surface charge prevents them from direct cell entry, they need to be packaged into specially designed delivery platforms to exert their activities. Multiple nanotechnology‐based delivery platforms have been developed for mRNA delivery, such as polymer‐based polyplex, lipid‐based lipoplex, and lipid‐coated polymer‐based lipopolyplex. In this review, an overview of the nanoplatforms for mRNA delivery is provided. A number of applications in biotechnology including cell reprogramming and gene editing are also described. In addition, their clinical translational potential for protein replacement therapy and for infectious disease and cancer treatment is introduced.

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Registry

Crossref · 10.1002/adtp.202000099

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237 of 237 references verified0 citation errors · 0 not checkable

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  • mRNA has therapeutic potential in viral vaccines, protein replacement therapies, cancer immunotherapies, cellular reprogramming and genome editing.

    “mRNA has shown therapeutic potential in a range of applications, including viral vaccines, protein replacement therapies, cancer immunotherapies, cellular reprogramming and genome editing2,4,7–17.”

    From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [14]

  • Lipid nanoparticle-mRNA formulations typically contain other lipid components, such as phospholipids, cholesterol or PEG-lipids.

    “In addition to cationic or ionizable lipids, lipid nanoparticle–mRNA formulations typically contain other lipid components, such as phospholipids (for example, phosphatidylcholine and phosphatidylethanolamine), cholesterol or polyethylene glycol (PEG)-functionalized lipids (PEG-lipids)7,14,17.”

    From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [14]

  • The pH-sensitivity of ionizable lipids is beneficial for mRNA delivery in vivo because neutral lipids have less interactions with anionic membranes of blood cells and improve biocompatibility.

    “The pH-sensitivity of ionizable lipids is beneficial for mRNA delivery in vivo, because neutral lipids have less interactions with the anionic membranes of blood cells and, thus, improve the biocompatibility of lipid nanoparticles7,11,14.”

    From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [14]

  • These other lipids can improve nanoparticle properties, such as particle stability, delivery efficacy, tolerability and biodistribution.

    “These lipids can improve nanoparticle properties, such as particle stability, delivery efficacy, tolerability and biodistribution7,14,17.”

    From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [14]

  • Trapped in endosomes, ionizable lipids are protonated and become positively charged, which may promote membrane destabilization and facilitate endosomal escape.

    “Trapped in endosomes, in which the pH is lower than in the extracellular environment, ionizable lipids are protonated and, therefore, become positively charged, which may promote membrane destabilization and facilitate endosomal escape of the nanoparticles7,11,14”

    From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [14]

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Authors
Meng, C., Chen, Z., Li, G., Welte, T., & Shen, H.
Affiliations
Houston Methodist (US)
Corresponding author
Shen, Haifa
Venue
Advanced Therapeutics
Publisher
Wiley
Published
2020-07-21
Volume
4
Issue
1
ISSN
2366-3987 (ISSN-L)
OpenAlex
W3045083782
Cited by
130
References
237
Citations by year
2026: 10 · 2025: 28 · 2024: 26 · 2023: 29 · 2022: 31 · 2021: 5 · 2020: 1
FWCI
5.35
Type
journal article
Language
en
Open access
closed
Locations
Advanced Therapeutics (publishedVersion)
Funders
National Institutes of Health (U54CA210181, R01CA193880, R01CA222959)
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
  • Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
  • Physical Sciences › Materials Science › Polymers and Plastics › Dendrimers and Hyperbranched Polymers
Keywords
mRNA therapeutics, mRNA delivery, nanoplatforms, polyplexes, lipoplexes, lipopolyplexes, in vitro transcribed mRNA, mRNA stability, cellular reprogramming, genome editing, protein replacement therapy, cancer therapy, infection treatment, clinical translation

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