Gene editing therapeutics based on mRNA delivery

Adv Drug Deliv Rev. 2023 Sep:200:115026. doi: 10.1016/j.addr.2023.115026. Epub 2023 Jul 27.

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.

Publication types

  • Review
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • CRISPR-Cas Systems
  • Gene Editing* / methods
  • Gene Transfer Techniques*
  • Genetic Therapy / methods
  • Humans
  • RNA, Messenger / genetics

Substances

  • RNA, Messenger