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Chromosomal effects of adeno-associated virus vector integration

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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Figure 1: Sequences of vector-chromosome junctions.
Figure 2: Chromosome 19 integration sites.

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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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Correspondence to David W. Russell.

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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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