Format

Send to

Choose Destination
Nat Med. 2013 Jul;19(7):889-91. doi: 10.1038/nm.3230. Epub 2013 Jun 16.

A largely random AAV integration profile after LPLD gene therapy.

Author information

1
National Center for Tumor Diseases and German Cancer Research Center, Heidelberg, Germany.

Abstract

The clinical application of adeno-associated virus vectors (AAVs) is limited because of concerns about AAV integration-mediated tumorigenicity. We performed integration-site analysis after AAV1-LPL(S447X) intramuscular injection in five lipoprotein lipase-deficient subjects, revealing random nuclear integration and hotspots in mitochondria. We conclude that AAV integration is potentially safe and that vector breakage and integration may occur from each position of the vector genome. Future viral integration-site analyses should include the mitochondrial genome.

PMID:
23770691
DOI:
10.1038/nm.3230
[Indexed for MEDLINE]

Supplemental Content

Full text links

Icon for Nature Publishing Group
Loading ...
Support Center