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◆ Molecular Therapy — Nucleic Acids2025-10-11· Genetic enhancement

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

Shengyi Wang, Xiaoyu Xu, Tapani Viitala, Yilai Shu, Hongbo Zhang

原始摘要(英文原文)· Original abstract
, 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.
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