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Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases

18 Jun 2024

Abstract excerpt

Liver-directed adeno-associated viral (AAV) vector-mediated homology-independent targeted integration (AAV-HITI) by CRISPR-Cas9 at the highly transcribed albumin locus is under investigation to provide sustained transgene expression following neonatal treatment. We show that targeting the 3' end of the albumin locus results in productive integration in about 15% of mouse hepatocytes achieving therapeutic levels...

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PMID: 38897206
DOI: 10.1016/j.xcrm.2024.101619

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