Efficient and accurate homologous recombination in hESCs and hiPSCs using helper-dependent adenoviral vectors

Mol Ther. 2012 Feb;20(2):424-31. doi: 10.1038/mt.2011.266. Epub 2011 Dec 6.

Abstract

Low efficiencies of gene targeting via homologous recombination (HR) have limited basic research and applications using human embryonic stem cells (hESCs) and human induced pluripotent stem cells (hiPSCs). Here, we show highly and equally efficient gene knockout and knock-in at both transcriptionally active (HPRT1, KU80, LIG1, LIG3) and inactive (HB9) loci in these cells using high-capacity helper-dependent adenoviral vectors (HDAdVs). Without the necessity of introducing artificial DNA double-strand breaks, 7-81% of drug-resistant colonies were gene-targeted by accurate HR, which were not accompanied with additional ectopic integrations. Even at the motor neuron-specific HB9 locus, the enhanced green fluorescent protein (EGFP) gene was accurately knocked in in 23-57% of drug-resistant colonies. In these clones, induced differentiation into the HB9-positive motor neuron correlated with EGFP expression. Furthermore, HDAdV infection had no detectable adverse effects on the undifferentiated state and pluripotency of hESCs and hiPSCs. These results suggest that HDAdV is one of the best methods for efficient and accurate gene targeting in hESCs and hiPSCs and might be especially useful for therapeutic applications.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenoviridae / genetics*
  • Antigens, Nuclear / genetics
  • Cell Line
  • DNA Ligase ATP
  • DNA Ligases / genetics
  • DNA-Binding Proteins / genetics
  • Embryonic Stem Cells / cytology
  • Embryonic Stem Cells / metabolism*
  • Gene Knock-In Techniques
  • Gene Knockout Techniques
  • Gene Order
  • Gene Targeting
  • Genetic Vectors / genetics*
  • Heterozygote
  • Homologous Recombination*
  • Humans
  • Hypoxanthine Phosphoribosyltransferase / genetics
  • Induced Pluripotent Stem Cells / cytology
  • Induced Pluripotent Stem Cells / metabolism*
  • Ku Autoantigen
  • Mutation
  • Poly-ADP-Ribose Binding Proteins
  • Xenopus Proteins

Substances

  • Antigens, Nuclear
  • DNA-Binding Proteins
  • LIG1 protein, human
  • Poly-ADP-Ribose Binding Proteins
  • Xenopus Proteins
  • Hypoxanthine Phosphoribosyltransferase
  • Xrcc6 protein, human
  • Ku Autoantigen
  • DNA Ligases
  • DNA Ligase ATP
  • DNA ligase III alpha protein, Xenopus
  • LIG3 protein, human