Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases

Nucleic Acids Res. 2012 Jul;40(13):6367-79. doi: 10.1093/nar/gks268. Epub 2012 Mar 29.

Abstract

The ability to specifically engineer the genome of living cells at precise locations using rare-cutting designer endonucleases has broad implications for biotechnology and medicine, particularly for functional genomics, transgenics and gene therapy. However, the potential impact of chromosomal context and epigenetics on designer endonuclease-mediated genome editing is poorly understood. To address this question, we conducted a comprehensive analysis on the efficacy of 37 endonucleases derived from the quintessential I-CreI meganuclease that were specifically designed to cleave 39 different genomic targets. The analysis revealed that the efficiency of targeted mutagenesis at a given chromosomal locus is predictive of that of homologous gene targeting. Consequently, a strong genome-wide correlation was apparent between the efficiency of targeted mutagenesis (≤ 0.1% to ≈ 6%) with that of homologous gene targeting (≤ 0.1% to ≈ 15%). In contrast, the efficiency of targeted mutagenesis or homologous gene targeting at a given chromosomal locus does not correlate with the activity of individual endonucleases on transiently transfected substrates. Finally, we demonstrate that chromatin accessibility modulates the efficacy of rare-cutting endonucleases, accounting for strong position effects. Thus, chromosomal context and epigenetic mechanisms may play a major role in the efficiency rare-cutting endonuclease-induced genome engineering.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • CHO Cells
  • Cell Line
  • Chromosomal Position Effects*
  • Cricetinae
  • Cricetulus
  • DNA Restriction Enzymes / chemistry
  • DNA Restriction Enzymes / metabolism*
  • Gene Targeting
  • Genetic Engineering
  • Genome, Human
  • Humans
  • Mutagenesis

Substances

  • DNA Restriction Enzymes
  • endodeoxyribonuclease CreI