基因组编辑
清脆的
基因传递
计算生物学
免疫原性
遗传增强
Cas9
生物
计算机科学
基因
纳米技术
遗传学
材料科学
抗原
作者
Fan Wu,Nei Li,Yi Xiao,Rohan Palanki,Hannah M. Yamagata,Michael J. Mitchell,Xuexiang Han
出处
期刊:Small methods
[Wiley]
日期:2025-05-28
卷期号:: e2401632-e2401632
被引量:7
标识
DOI:10.1002/smtd.202401632
摘要
Abstract Gene editing has emerged as a promising therapeutic option for treating genetic diseases. However, a central challenge in the field is the safe and efficient delivery of these large editing tools, especially in vivo. Lipid nanoparticles (LNPs) are attractive nonviral vectors due to their low immunogenicity and high delivery efficiency. To maximize editing efficiency, LNPs should efficiently protect gene editing components against multiple biological barriers and release them into the cytoplasm of target cells. In this review, the widely used CRISPR gene editing systems are first overviewed. Then, each component of LNPs, as well as their effects on delivery, are systematically discussed. Following this, the current LNP engineering strategies to achieve non‐liver targeting are summarized. Finally, preclinical and clinical applications of LNPs for in vivo genome editing are highlighted, and perspectives for the future development of LNPs are provided.
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