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Cas12a chRDNA-mediated in vivo genome editing for high specificity functional gene disruption

CRISPR hybrid guides containing RNA and DNA nucleotides (chRDNA) enhance Cas nuclease specificity by reducing off-target editing. Intravenous administration of LNP-encapsulated Cas12a mRNA with a chRDNA guide in mice led to dose-responsive hepatic genome editing without detectable off-target editing and near elimination of the targeted plasma protein for one year.
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