Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2023 Oct 2;25(6):95.
doi: 10.1208/s12248-023-00860-z.

RNA Nanomedicine: Delivery Strategies and Applications

Affiliations
Review

RNA Nanomedicine: Delivery Strategies and Applications

Junho Byun et al. AAPS J. .

Abstract

Delivery of RNA using nanomaterials has emerged as a new modality to expand therapeutic applications in biomedical research. However, the delivery of RNA presents unique challenges due to its susceptibility to degradation and the requirement for efficient intracellular delivery. The integration of nanotechnologies with RNA delivery has addressed many of these challenges. In this review, we discuss different strategies employed in the design and development of nanomaterials for RNA delivery. We also highlight recent advances in the pharmaceutical applications of RNA delivered via nanomaterials. Various nanomaterials, such as lipids, polymers, peptides, nucleic acids, and inorganic nanomaterials, have been utilized for delivering functional RNAs, including messenger RNA (mRNA), small interfering RNA, single guide RNA, and microRNA. Furthermore, the utilization of nanomaterials has expanded the applications of functional RNA as active pharmaceutical ingredients. For instance, the delivery of antigen-encoding mRNA using nanomaterials enables the transient expression of vaccine antigens, leading to immunogenicity and prevention against infectious diseases. Additionally, nanomaterial-mediated RNA delivery has been investigated for engineering cells to express exogenous functional proteins. Nanomaterials have also been employed for co-delivering single guide RNA and mRNA to facilitate gene editing of genetic diseases. Apart from the progress made in RNA medicine, we discuss the current challenges and future directions in this field.

Keywords: RNA delivery; RNA nanomedicine; RNA therapeutics; RNA vaccine; nanomaterials.

PubMed Disclaimer

References

    1. Barbier AJ, Jiang AY, Zhang P, Wooster R, Anderson DG. The clinical progress of mRNA vaccines and immunotherapies. Nat Biotechnol. 2022;40(6):840–54. - PubMed
    1. Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, et al. Post-transcriptional genetic silencing of BCL11A to treat sickle cell disease. N Engl J Med. 2021;384(3):205–15. - PubMed
    1. Wang C, Zhang Y, Dong Y. Lipid nanoparticle-mRNA formulations for therapeutic applications. Acc Chem Res. 2021;54(23):4283–93. - PubMed - PMC
    1. Sabnis S, Kumarasinghe ES, Salerno T, Mihai C, Ketova T, Senn JJ, et al. A novel amino lipid series for mRNA delivery: improved endosomal escape and sustained pharmacology and safety in non-human primates. Mol Ther. 2018;26(6):1509–19. - PubMed - PMC
    1. Frangoul H, Altshuler D, Cappellini D, Chen YS, Domm J, Eustace BK, et al. CRISPR-Cas9 gene editing for sickle cell disease and beta-thalassemia. N Engl J Med. 2021;384(23): e91. - PubMed

Publication types

LinkOut - more resources