Article

Highly Efficient Functional Editing of the Native Ornithine Transcarbamylase Locus by Targeted Integration with Phenotype Correction and Restoration of Physiological Patterns of Gene Expression

2025-08-08

Abstract excerpt

<title>Abstract</title> <p>Here we report unprecedented efficacy in the functional repair of an exemplar locus, ornithine transcarbamylase (OTC), in mutant primary mouse and human hepatocytes in vivo using a dual AAV vector system configured to deliver CRISPR-Cas9 editing reagents and a promoterless donor for targeted integration by non-homologous end joining. The approach was mutation agnostic and targeted editi...

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Literature Corpus work: dde2c068-878b-57fe-ba4f-e76f4793aa9c
DOI: 10.21203/rs.3.rs-7237670/v1

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