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

A valuable paper examining the presence of AAV manufacturing contaminants in a human patient’s liver. These results will hopefully be leveraged for improving the safety of gene therapies in the future.


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.

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