Foamy viral vector integration sites in SCID-repopulating cells after MGMTP140K-mediated in vivo selection

Gene Ther. 2015 Jul;22(7):591-5. doi: 10.1038/gt.2015.20. Epub 2015 Mar 19.

Abstract

Foamy virus (FV) vectors are promising for hematopoietic stem cell (HSC) gene therapy but preclinical data on the clonal composition of FV vector-transduced human repopulating cells is needed. Human CD34(+) human cord blood cells were transduced with an FV vector encoding a methylguanine methyltransferase (MGMT)P140K transgene, transplanted into immunodeficient NOD/SCID IL2Rγ(null) mice, and selected in vivo for gene-modified cells. The retroviral insertion site profile of repopulating clones was examined using modified genomic sequencing PCR. We observed polyclonal repopulation with no evidence of clonal dominance even with the use of a strong internal spleen focus forming virus promoter known to be genotoxic. Our data supports the use of FV vectors with MGMTP140K for HSC gene therapy but also suggests additional safety features should be developed and evaluated.

Publication types

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

MeSH terms

  • Animals
  • Genetic Testing / methods
  • Genetic Vectors
  • Hematopoietic Stem Cell Transplantation
  • Hematopoietic Stem Cells
  • Humans
  • Mice, Inbred NOD
  • Mice, SCID
  • Severe Combined Immunodeficiency / genetics*
  • Spumavirus / genetics*
  • Transplantation Conditioning
  • Virus Integration / genetics*