Frequent endonuclease cleavage at off-target locations in vivo

Mol Ther. 2010 May;18(5):983-6. doi: 10.1038/mt.2010.35. Epub 2010 Mar 9.

Abstract

Target-site DNA breaks increase recombination frequencies, however, the specificity of the enzymes used to create them remains poorly defined. The location and frequency of off-target cleavage events are especially important when rare-cutting endonucleases are used in clinical settings. Here, we identify noncanonical cleavage sites of I-SceI that are frequently cut in the human genome by localizing adeno-associated virus (AAV) vector-chromosome junctions, demonstrating the importance of in vivo characterization of enzyme cleavage specificity.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Cell Line
  • DNA Breaks, Double-Stranded
  • Dependovirus / genetics
  • Endonucleases / metabolism*
  • Genetic Vectors / genetics
  • Humans

Substances

  • Endonucleases