Foamy-virus-mediated gene transfer to canine repopulating cells

Blood. 2007 Jan 1;109(1):65-70. doi: 10.1182/blood-2006-04-016741. Epub 2006 Sep 12.

Abstract

Foamy virus (FV) vectors are particularly attractive gene-transfer vectors for stem-cell gene therapy because they form a stable transduction intermediate in quiescent cells and can efficiently transduce hematopoietic stem cells. Here, we studied the use of FV vectors to transduce long-term hematopoietic repopulating cells in the dog, a clinically relevant large animal model. Mobilized canine peripheral blood (PB) CD34+ cells were transduced with an enhanced green fluorescent protein (EGFP)-expressing FV vector in an 18-hour transduction protocol. All 3 dogs studied had rapid neutrophil engraftment to greater than 500/microL with a median of 10 days. Transgene expression was detected in all cell lineages (B cells, T cells, granulocytes, red blood cells, and platelets), indicating multilineage engraftment of transduced cells. Up to 19% of blood cells were EGFP+, and this was confirmed at the DNA level by real-time polymerase chain reaction (PCR) and Southern blot analysis. These transduction rates were higher than the best results we obtained previously with lentiviral vectors in a similar transduction protocol. Integration site analysis also demonstrated polyclonal repopulation and the transduction of multipotential hematopoietic repopulating cells. These data suggest that FV vectors should be useful for stem-cell gene therapy, particularly for applications in which short transduction protocols are critical.

Publication types

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

MeSH terms

  • Animals
  • Blood Cells / cytology
  • Blood Cells / metabolism
  • Blotting, Southern
  • Cell Lineage
  • Colony-Forming Units Assay
  • Dogs
  • Genes, Reporter
  • Genetic Vectors / genetics*
  • Graft Survival
  • Green Fluorescent Proteins / analysis
  • Green Fluorescent Proteins / biosynthesis
  • Green Fluorescent Proteins / genetics
  • Hematopoietic Stem Cells / virology*
  • Mice
  • Peripheral Blood Stem Cell Transplantation*
  • Phosphoglycerate Kinase / genetics
  • Polymerase Chain Reaction
  • Radiation Chimera
  • Spumavirus / genetics*
  • Transduction, Genetic*
  • Transgenes
  • Transplantation, Autologous

Substances

  • enhanced green fluorescent protein
  • Green Fluorescent Proteins
  • Phosphoglycerate Kinase