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.
使用 AI 将内容摘要翻译为中文,便于快速阅读
使用 AI 分析这篇文章的核心发现、关键要点和深度见解
由 DeepSeek AI 提供分析 · 首次使用需配置 API Key
arXiv · 2006-03-27
arXiv · 2024-03-06
arXiv · 2021-08-10
arXiv · 2018-07-03
arXiv · 2019-03-13