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 Sep:200:115026.
doi: 10.1016/j.addr.2023.115026. Epub 2023 Jul 27.

Gene editing therapeutics based on mRNA delivery

Juliana Popovitz  1 , Rohit Sharma  2 , Reyhane Hoshyar  1 , Beob Soo Kim  1 , Niren Murthy  3 , Kunwoo Lee  4
Affiliations
Review

Gene editing therapeutics based on mRNA delivery

Juliana Popovitz et al. Adv Drug Deliv Rev. 2023 Sep.

Abstract

The field of gene editing has received much attention in recent years due to its immense therapeutic potential. In particular, gene editing therapeutics, such as the CRISPR-Cas systems, base editors, and other emerging gene editors, offer the opportunity to address previously untreatable disorders. This review aims to summarize the therapeutic applications of gene editing based on mRNA delivery. We introduce gene editing therapeutics using mRNA and focus on engineering and improvement of gene editing technology. We subsequently examine ex vivo and in vivo gene editing techniques and conclude with an exploration of the next generation of CRISPR and base editing systems.

Keywords: Base editing; CRISPR; Delivery; Gene editing; Gene therapy; Nanoparticle; mRNA.

PubMed Disclaimer

Conflict of interest statement

Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Publication types

LinkOut - more resources