Unlocking mRNA-driven CRISPR-Cas9 gene therapy via optimizing mRNA and the delivery vectors

作者：Shengyi Wang, Xiaoyu Xu, Tapani Viitala, Yilai Shu, Hongbo Zhang · 发表于：Molecular Therapy — Nucleic Acids · 年份：2025 · DOI：10.1016/j.omtn.2025.102737 · 被引用次数：5 · 研究领域：CRISPR and Genetic Engineering、RNA Interference and Gene Delivery、Pluripotent Stem Cells Research

, and off-target effects. This review summarizes recent advancements in efforts to enhance mRNA stability and translation efficiency, and it describes novel delivery vectors currently being used for mRNA-driven CRISPR-Cas9 therapies. Moreover, the development of novel gene editors based on CRISPR-Cas9 engineering and the development of length fragment integration technology based on prime editing tool engineering are also discussed. The discussion of these advances aims to provide a full picture of the challenges of mRNA-based CRISPR-Cas9 therapeutics for the treatment of various diseases.