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Adeno-associated virus finds its disease

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Adeno-associated virus (AAV) vectors have been widely adopted for use in gene therapy. A new study raises concerns regarding this approach, reporting that chromosomal insertions of AAV serotype 2 seem to activate proto-oncogenes in human hepatocellular carcinoma.

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Figure 1: AAV and hepatocellular carcinoma.

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Correspondence to David W Russell or Markus Grompe.

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Scientific Advisory Board, Horizon Discovery. Equity in Universal Cells, Inc.

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Russell, D., Grompe, M. Adeno-associated virus finds its disease. Nat Genet 47, 1104–1105 (2015). https://doi.org/10.1038/ng.3407

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