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通过优化mRNA和传递载体来解锁mRNA驱动的CRISPR-Cas9基因疗法

Shengyi Wang^1,2,3, Xiaoyu Xu^2,3,4,5, Tapani Viitala^3,6

¹Joint Centre of Translational Medicine, The First Affiliated Hospital of Wenzhou Medical University, Wenzhou, Zhejiang 325015, China.

Molecular therapy. Nucleic acids

|November 10, 2025

概括

由信使RNA (mRNA) 驱动的CRISPR-Cas9基因疗法可以在没有基因组整合的情况下提供高效的编辑. 本综述涵盖了mRNA稳定性,传递和新型基因编辑器的进展,以克服临床挑战.

科学领域:

分子生物学分子生物学
基因治疗基因治疗
生物技术是生物技术.

背景情况:

基于mRNA的CRISPR-Cas9基因疗法对高效的基因组编辑有希望.
目前的限制包括mRNA不稳定性,免疫性,短半衰期和低效的体内输送.
非目标效应也对临床应用构成重大挑战.

研究的目的:

审查mRNA驱动的CRISPR-Cas9基因疗法的最新进展.
突出提高mRNA稳定性和翻译效率的策略.
讨论新的传递载体和基因编辑工具.

主要方法:

关于基于mRNA的基因编辑的最新研究的文献综述.
对CRISPR-Cas9和原始编辑技术的进展进行分析.
检查用于mRNA治疗的新型输送系统.

主要成果:

在提高mRNA稳定性和翻译效率方面取得了进展.
新的传递载体正在开发中,以增强体内传递.
新的基因编辑器和主要编辑技术显示出提高准确性和效率的潜力.

结论:

克服mRNA稳定性,分发和特异性的挑战对于临床翻译至关重要.
基因编辑工具和传递系统的持续创新将推动基于mRNA的疗法.
本综述为mRNA驱动的CRISPR-Cas9疗法提供了当前景观和未来方向的全面概述.

关键词:

这就是CRISPR-Cas9的特征.MT:RNA/DNA编辑编辑交付载体的交付载体工程到mRNA的工程.基因治疗的基因疗法长度碎片集成技术技术的整合

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