Cited by 389RNA Interference and Gene DeliveryNanoparticle-Based Drug DeliveryAdvanced Drug Delivery Systems

Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems

Guan, S, Rosenecker, J·2017·Gene Therapy

Abstract

Because of its safe and effective protein expression profile, in vitro transcribed messenger RNA (IVT-mRNA) represents a promising candidate in the development of novel therapeutics for genetic diseases, vaccines or gene editing strategies, especially when its inherent shortcomings (for example, instability and immunogenicity) have been partially addressed via structural modifications. However, numerous unsolved technical difficulties in successful in vivo delivery of IVT-mRNA have greatly hindered the applications of IVT-mRNA in clinical development. Recent advances in nanotechnology and material science have yielded many promising nonviral delivery systems, some of which were able to efficiently facilitate targeted in vivo delivery of IVT-mRNA in safe and noninvasive manners. The diversity and flexibility of these delivery systems highlight the recent progress of IVT-mRNA-based therapy using nonviral vectors. In this review, we summarize recent advances of existing and emerging nonviral vector-based nanotechnologies for IVT-mRNA delivery and briefly summarize the interesting but rarely discussed applications on simultaneous delivery of IVT-mRNA with DNA.

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Crossref · 10.1038/gt.2017.5

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122 of 122 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 [9]

  • Engineering of mRNA molecules and design of lipid nanoparticles have been optimized, contributing to the rapid development and clinical assessment of the two COVID-19 mRNA vaccines.

    “To address these concerns, the engineering of mRNA molecules (Box 1) and the design of lipid nanoparticles have been optimized2,4,7–17, which has contributed to the rapid development and clinical assessment15,17 of the two COVID-19 mRNA vaccines, mRNA-1273 (NCT04470427)19,20 and BNT162b2 (NCT04368728)21.”

    From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [9]

  • Polymers such as polyethyleneimine, protamine and polyaspartamide derivatives are already widely used for mRNA delivery.

    “Polymers, such as polyethyleneimine, protamine and polyaspartamide derivatives, are already widely used for mRNA delivery7–17.”

    From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [9]

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Authors
Guan, S., & Rosenecker, J.
Affiliations
Ludwig-Maximilians-Universität München (DE)
Corresponding author
Rosenecker, J
Venue
Gene Therapy
Published
2017-01-17
Volume
24
Issue
3
Pages
133-143
ISSN
  • 0969-7128 (ISSN-L)
  • 1476-5462
OpenAlex
W2573033399
Cited by
389
References
122
Citations by year
2026: 18 · 2025: 45 · 2024: 49 · 2023: 40 · 2022: 65 · 2021: 59 · 2020: 39 · 2019: 32 · 2018: 35 · 2017: 7
FWCI
2.89
Type
journal article
Language
en
Open access
closed
Locations
  • Gene Therapy (publishedVersion)
  • PubMed (publishedVersion)
Funders
China Scholarship Council
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
  • Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
  • Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advanced Drug Delivery Systems
Keywords
mRNA therapeutics, in vitro transcribed mRNA, non-viral vectors, mRNA delivery, nanotechnology, targeted delivery, mRNA stability, immunogenicity, genome editing, vaccines, genetic disorders
MeSH terms
Animals, Humans, RNA, Messenger, Genetic Therapy, Gene Transfer Techniques, Nanotechnology, Nanostructures

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