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

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Abstract

RNA forms of CRISPR-Cas9-based gene therapies provide more safer and effective therapeutic outcomes. This paper offers a reference for improving the effects of RNA forms of CRISPR-Cas9-based therapies, through the rational engineering design of mRNA components, combined with novel structural RNAs and novel delivery vehicles.

Original languageEnglish
Article number102737
JournalMolecular Therapy Nucleic Acids
Volume36
Issue number4
DOIs
Publication statusPublished - Dec 2025
MoE publication typeA2 Review article in a scientific journal

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