Gene editing of human embryonic stem cells via an engineered baculoviral vector carrying zinc-finger nucleases

Mol Ther. 2011 May;19(5):942-50. doi: 10.1038/mt.2011.12. Epub 2011 Feb 15.

Abstract

Human embryonic stem (hES) cells are renewable cell sources that have potential applications in regenerative medicine. The development of technologies to produce permanent and site-specific genome modifications is in demand to achieve future medical implementation of hES cells. We report herein that a baculoviral vector (BV) system carrying zinc-finger nucleases (ZFNs) can successfully modify the hES cell genome. BV-mediated transient expression of ZFNs specifically disrupted the CCR5 locus in transduced cells and the modified cells exhibited resistance to HIV-1 transduction. To convert the BV to a gene targeting vector, a DNA donor template and ZFNs were incorporated into the vector. These hybrid vectors yielded permanent site-specific gene addition in both immortalized human cell lines (10%) and hES cells (5%). Modified hES cells were both karyotypically normal and pluripotent. These results suggest that this baculoviral delivery system can be engineered for site-specific genetic manipulation in hES cells.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Line
  • Deoxyribonucleases / genetics*
  • Deoxyribonucleases / metabolism
  • Embryonic Stem Cells / metabolism*
  • Flow Cytometry
  • Gene Targeting
  • Genetic Vectors
  • HIV-1 / genetics
  • Humans
  • Mutation
  • Nucleopolyhedroviruses / genetics*
  • Receptors, CCR5 / genetics*
  • Reverse Transcriptase Polymerase Chain Reaction
  • Transduction, Genetic
  • Transgenes
  • Zinc Fingers

Substances

  • Receptors, CCR5
  • Deoxyribonucleases