基于 mRNA 递送的基因编辑治疗学。

Gene editing therapeutics based on mRNA delivery.

机构信息

GenEdit, 681 Gateway Blvd., South San Francisco, CA 94080, USA.

Department of Bioengineering, University of California, Berkeley, CA 94720, USA; Innovative Genomics Institute, 2151 Berkeley Way, Berkeley, CA 94704, USA.

出版信息

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

DOI:10.1016/j.addr.2023.115026

PMID:37516409

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.

摘要

近年来，由于其巨大的治疗潜力，基因编辑领域受到了广泛关注。特别是基因编辑疗法，如 CRISPR-Cas 系统、碱基编辑器和其他新兴的基因编辑工具，为治疗以前无法治疗的疾病提供了机会。本综述旨在总结基于 mRNA 递送的基因编辑的治疗应用。我们介绍了使用 mRNA 的基因编辑疗法，并重点介绍了基因编辑技术的工程和改进。随后，我们研究了体外和体内基因编辑技术，并探讨了下一代 CRISPR 和碱基编辑系统。