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AAV capsid prioritization in normal and steatotic human livers maintained by machine perfusion

29 Jan 2025

Abstract excerpt

Therapeutic efficacy and safety of adeno-associated virus (AAV) liver gene therapy depend on capsid choice. To predict AAV capsid performance under near-clinical conditions, we established side-by-side comparison at single-cell resolution in human livers maintained by normothermic machine perfusion. AAV-LK03 transduced hepatocytes much more efficiently and specifically than AAV5, AAV8 and AAV6, which are most...

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PMID: 39881029
DOI: 10.1038/s41587-024-02523-6

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