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This study aims to investigate the presence of plasmid sequences and the degree of vector genome disruption in the liver after AAV gene therapy.

January 18, 2026Nature MedicineOpen Access

Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy

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Case study highlights liver toxicity mechanisms in a child post-AAV gene therapy, suggesting critical implications for future treatments.

This study aims to investigate the presence of plasmid sequences and the degree of vector genome disruption in the liver after AAV gene therapy.
Conducted metagenomic sequencing of liver tissue post-gene therapy.
Evaluated the presence of manufacturing plasmid sequences and helper viruses.
Analyzed the structure and integrity of vector genomes.
Identified complex plasmid structures and extensive vector genome disruption.
Found numerous vector–human fusion junctions in the liver.
Detected human betaherpesvirus 6B in the liver tissue.

Buddle et al. (2026) studied this question.

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