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Engineered PsCas9 enables therapeutic genome editing in mouse liver with lipid nanoparticles

7 Nov 2024

Abstract excerpt

Clinical implementation of therapeutic genome editing relies on efficient in vivo delivery and the safety of CRISPR-Cas tools. Previously, we identified PsCas9 as a Type II-B family enzyme capable of editing mouse liver genome upon adenoviral delivery without detectable off-targets and reduced chromosomal translocations. Yet, its efficacy remains insufficient with non-viral delivery, a common challenge for many...

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PMID: 39511150
DOI: 10.1038/s41467-024-53418-8

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