Gene editing therapeutics based on mRNA delivery

被引:15
|
作者
Popovitz, Juliana [1 ]
Sharma, Rohit [2 ,3 ]
Hoshyar, Reyhane [1 ]
Kim, Beob Soo [1 ]
Murthy, Niren [2 ,3 ]
Lee, Kunwoo [1 ]
机构
[1] GenEdit, 681 Gateway Blvd, San Francisco, CA 94080 USA
[2] Univ Calif Berkeley, Dept Bioengn, Berkeley, CA 94720 USA
[3] Innovat Genom Inst, 2151 Berkeley Way, Berkeley, CA 94704 USA
关键词
Gene therapy; CRISPR; mRNA; Nanoparticle; Delivery; Gene editing; Base editing; NANOPARTICLE DELIVERY; LIPID NANOPARTICLES; SYSTEMIC DELIVERY; GUIDE RNA; T-CELLS; THERAPY; CRISPR; OPTIMIZATION; TRANSFECTION; TRANSLATION;
D O I
10.1016/j.addr.2023.115026
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
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.
引用
收藏
页数:12
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