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Directed evolution of liver-detargeted AAV vectors for systemic gene delivery to skeletal muscle and heart

21 Aug 2025

Abstract excerpt

Adeno-associated virus (AAV) gene therapies that require systemic administration of high vector doses are associated with hepatotoxicity risk, given the liver tropism of most AAV vectors. We combined screening of natural serotypes, targeted mutagenesis, and directed evolution to identify liver-detargeted muscle-tropic capsids. We found that AAVhu.32 is liver-detargeted compared to AAV9, and AAV5 exhibits high...

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RNA Interference and Gene Delivery
Tissue Engineering and Regenerative Medicine
Virus-based gene therapy research

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 40989879
DOI: 10.1016/j.omtm.2025.101571

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