Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy
16 January 2026 at 08:00
Nature Medicine, Published online: 16 January 2026; doi:10.1038/s41591-025-04073-z
Analyses of liver biopsies from a child with spinal muscular atrophy treated with adeno-associated virus gene therapy who developed hepatitis reveal contaminating manufacturing plasmids and disrupted vector genomes, possibly resulting from recombination events.