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
. 2021 Nov:178:113834.
doi: 10.1016/j.addr.2021.113834. Epub 2021 Sep 4.

Nucleic acid delivery for therapeutic applications

Akash Gupta  1 , Jason L Andresen  2 , Rajith S Manan  3 , Robert Langer  4
Affiliations
Review

Nucleic acid delivery for therapeutic applications

Akash Gupta et al. Adv Drug Deliv Rev. 2021 Nov.

Abstract

Recent medical advances have exploited the ability to address a given disease at the underlying level of transcription and translation. These treatment paradigms utilize nucleic acids - including short interfering RNA (siRNA), microRNA (miRNA), antisense oligonucleotides (ASO), and messenger RNA (mRNA) - to achieve a desired outcome ranging from gene knockdown to induced expression of a selected target protein. Towards this end, numerous strategies for encapsulation or stabilization of various nucleic acid structures have been developed in order to achieve intracellular delivery. In this review, we discuss several therapeutic applications of nucleic acids directed towards specific diseases and tissues of interest, in particular highlighting recent technologies which have reached late-stage clinical trials and received FDA approval.

Keywords: Biomaterials; COVID-19; Cancer; Genetic disease; Nucleic acid (oligonucleotide) delivery; Therapeutics; Vaccines.

PubMed Disclaimer

Conflict of interest statement

Declaration of Competing Interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: R.L. is a founder of Moderna and a founding scientific advisory board member of Alnylam Pharmaceuticals. For a list of entities with which R.L. is involved, compensated or uncompensated, see www.dropbox.com/s/yc3xqb5s8s94v7x/Rev%20Langer%20COI.pdf?dl = 0. A.G, J.L.A, and R.S.M, have no conflicts or competing interests to disclose.

Publication types

MeSH terms

LinkOut - more resources