From the RNA world to the clinic

Sullenger, Bruce A.; Nair, Smita

doi:10.1126/science.aad8709

Cited by 276RNA Interference and Gene DeliveryRNA and protein synthesis mechanismsRNA Research and Splicing

From the RNA world to the clinic

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Sullenger, Bruce A., Nair, Smita·2016·Science

DOI 10.1126/science.aad8709PMC PMC6035743

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Abstract

The study of RNA has continually emphasized the structural and functional versatility of RNA molecules. This versatility has inspired translational and clinical researchers to explore the utility of RNA-based therapeutic agents for a wide variety of medical applications. Several RNA therapeutics, with diverse modes of action, are being evaluated in large late-stage clinical trials, and many more are in early clinical development. Hundreds of patients are enrolled in large trials testing messenger RNAs to combat cancer, small interfering RNAs to treat renal and hepatic disorders, and aptamers to combat ocular and cardiovascular disease. Results from these studies are generating considerable interest among the biomedical community and the public and will be important for the future development of this emerging class of therapeutic agents.

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Vaccines based on mRNA offer multifaceted advantages including flexible antigen design, a cost-effective manufacturing process allowing for parallel production of multiple mRNA vaccines, and rapid manufacturing.
“Vaccines based on mRNA could meet these requirements because they offer multifaceted advantages including flexible antigen design, a cost-effective manufacturing process allowing for parallel production of multiple mRNA vaccines1–5 and rapid manufacturing, which could be of crucial importance during pandemics.6”
From Unmodified mRNA in LNPs constitutes a competitive technology for prophylactic vaccines (2017) · cites this paper as [3]
The “Therapeutic coding RNAs: mRNA-based immunotherapy” section describes mRNA vaccines encoding different tumor or viral antigens and says direct-injection approaches could replace more complicated and costly cell-based therapies. It does not establish a cost-effective manufacturing process or parallel production of multiple vaccines. Rapid manufacturing is attached to a separate citation in the citing sentence. (LLM, conf=0.83)
Experimental — accuracy not yet measured
RNA vaccines have exhibited encouraging results in both infection therapy and cancer immunotherapy.
“RNA vaccines, for example, have exhibited encouraging results in both infection therapy and cancer immunotherapy.[6, 7, 11–15]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [11]
RNA interference technology enables downregulation of disease-related genes and protein expression.
“For instance, RNA interference (RNAi) technology enables the downregulation of disease related genes and protein expression; mRNA mediates the expression and supplementation of therapeutic proteins; the CRISPR gene editing system facilitates gene engineering in vivo or through cell engineering ex vivo.[6, 9, 11, 17–20]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [11]
mRNA mediates the expression and supplementation of therapeutic proteins.
“For instance, RNA interference (RNAi) technology enables the downregulation of disease related genes and protein expression; mRNA mediates the expression and supplementation of therapeutic proteins; the CRISPR gene editing system facilitates gene engineering in vivo or through cell engineering ex vivo.[6, 9, 11, 17–20]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [11]
The CRISPR gene editing system facilitates gene engineering in vivo or through cell engineering ex vivo.
“For instance, RNA interference (RNAi) technology enables the downregulation of disease related genes and protein expression; mRNA mediates the expression and supplementation of therapeutic proteins; the CRISPR gene editing system facilitates gene engineering in vivo or through cell engineering ex vivo.[6, 9, 11, 17–20]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [11]

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Authors: Sullenger, B. A., & Nair, S.
Affiliations: Duke University (US)
Corresponding author: Sullenger, Bruce A.
ORCID: Sullenger, Bruce A.
Nair, Smita
Venue: Science
Publisher: American Association for the Advancement of Science (AAAS)
Published: 2016-06-17
Volume: 352
Issue: 6292
Pages: 1417-1420
ISSN: 0036-8075 (ISSN-L)
1095-9203
DOI: 10.1126/science.aad8709
PMC: PMC6035743
OpenAlex: W2434872344
Cited by: 274
References: 40
Citations by year: 2026: 7 · 2025: 18 · 2024: 29 · 2023: 23 · 2022: 34 · 2021: 33 · 2020: 32 · 2019: 28 · 2018: 30 · 2017: 34 · 2016: 6
FWCI: 2.99
Type: journal article
Language: en
Open access: green
europepmc.org
Locations: Science (publishedVersion)
PubMed (publishedVersion)
Europe PMC (PubMed Central) (acceptedVersion)
PubMed Central (acceptedVersion)
Funders: NIH
Department of Defense
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA and protein synthesis mechanisms
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Research and Splicing
Keywords: RNA therapeutics, mRNA, siRNA, aptamers, clinical trials, cancer therapy, kidney disease, liver disease, eye diseases, cardiovascular disease
MeSH terms: Cellular Reprogramming Techniques, Animals, Biological Therapy, Humans, Immunotherapy, Proteins, RNA, Messenger, Protein Biosynthesis, Cancer Vaccines, RNA, Small Interfering, RNA, Long Noncoding

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