Viral and Synthetic RNA Vector Technologies and Applications

Schott, Juliane W; Morgan, Michael; Galla, Melanie; Schambach, Axel

doi:10.1038/mt.2016.143

Cited by 76RNA Interference and Gene DeliveryVirus-based gene therapy researchRNA Research and Splicing

Viral and Synthetic RNA Vector Technologies and Applications

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Schott, Juliane W, Morgan, Michael, Galla, Melanie, Schambach, Axel·2016·Molecular Therapy

DOI 10.1038/mt.2016.143PMC PMC5113109

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Abstract

Use of RNA is an increasingly popular method to transiently deliver genetic information for cell manipulation in basic research and clinical therapy. In these settings, viral and nonviral RNA platforms are employed for delivery of small interfering RNA and protein-coding mRNA. Technological advances allowing RNA modification for increased stability, improved translation and reduced immunogenicity have led to increased use of nonviral synthetic RNA, which is delivered in naked form or upon formulation. Alternatively, highly efficient viral entry pathways are exploited to transfer genes of interest as RNA incorporated into viral particles. Current viral RNA transfer technologies are derived from Retroviruses, nonsegmented negative-strand RNA viruses or positive-stranded Alpha- and Flaviviruses. In retroviral particles, the genes of interest can either be incorporated directly into the viral RNA genome or as nonviral RNA. Nonsegmented negative-strand virus-, Alpha- and Flavivirus-derived vectors support prolonged expression windows through replication of viral RNA encoding genes of interest. Mixed technologies combining viral and nonviral components are also available. RNA transfer is ideal for all settings that do not require permanent transgene expression and excludes potentially detrimental DNA integration into the target cell genome. Thus, RNA-based technologies are successfully applied for reprogramming, transdifferentiation, gene editing, vaccination, tumor therapy, and gene therapy.

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Engineered saRNA vaccines rely on the insertion of the gene encoding the target antigen into the genome of an RNA virus (mainly alphavirus) as well as the use of its replication machinery to amplify the delivered RNA, thereby increasing antigen expression.
“Engineered saRNA vaccines rely on the insertion of the gene encoding the target antigen into the genome of an RNA virus (mainly alphavirus) as well as the use of its replication machinery to amplify the delivered RNA, thereby increasing antigen expression.191–193”
From Advances in COVID-19 mRNA vaccine development (2022) · cites this paper as [193]

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Authors: Schott, J. W., Morgan, M., Galla, M., & Schambach, A.
Affiliations: Medizinische Hochschule Hannover (DE)
Boston Children's Hospital (US)
Corresponding author: Schambach, Axel
ORCID: Schott, Juliane W
Galla, Melanie
Schambach, Axel
Venue: Molecular Therapy
Publisher: Elsevier BV
Published: 2016-09
Volume: 24
Issue: 9
Pages: 1513-1527
ISSN: 1525-0016 (ISSN-L)
DOI: 10.1038/mt.2016.143
PMC: PMC5113109
OpenAlex: W2464076669
Cited by: 76
References: 199
Citations by year: 2026: 4 · 2025: 7 · 2024: 7 · 2023: 13 · 2022: 8 · 2021: 12 · 2020: 7 · 2019: 6 · 2018: 6 · 2017: 5 · 2016: 1
FWCI: 0.54
Type: journal article
Language: en
Open access: hybrid
doi.org
Licence: cc-by-nc-nd
www.elsevier.com/tdm/userlicense/1.0/ (tdm)
creativecommons.org/licenses/by-nc-nd/4.0/ (vor, 365 d)
Locations: Molecular Therapy (publishedVersion, cc-by-nc-nd)
PubMed Central (publishedVersion)
PubMed (publishedVersion)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Research and Splicing
Keywords: synthetic RNA, siRNA, mRNA delivery, retroviral vectors, nonsegmented negative-strand RNA viruses, alphavirus, flavivirus, RNA modification, transient expression, cellular reprogramming, transdifferentiation, genome editing, vaccination, cancer therapy, gene therapy
MeSH terms: Animals, Biotechnology, Gene Expression Regulation, Genetic Engineering, Genetic Vectors, Humans, RNA, Virus Replication, Viruses, Gene Transfer Techniques

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