Abstract
Adeno-associated virus (AAV) vectors are currently being used in several clinical gene-therapy trials (see the NIH OBA Human Gene Transfer Clinical Trials Database); however, little is known about the chromosomal effects of vector integration. Here we report that integrated vector proviruses are associated with chromosomal deletions and other rearrangements and are frequently located on chromosome 19 (although not at the wildtype AAV integration site).
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Acknowledgements
We thank R. Dong, R. Newton and the Seattle Children's Hospital cytogenetics laboratory for technical assistance. This work was supported by grants from the Cystic Fibrosis Foundation, the March of Dimes and the National Institutes of Health.
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Miller, D., Rutledge, E. & Russell, D. Chromosomal effects of adeno-associated virus vector integration. Nat Genet 30, 147–148 (2002). https://doi.org/10.1038/ng824
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DOI: https://doi.org/10.1038/ng824
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