Transduction of long-term and mobilized peripheral blood-derived NOD/SCID repopulating cells by foamy virus vectors

Hum Gene Ther. 2004 Jan;15(1):87-92. doi: 10.1089/10430340460732481.

Abstract

Foamy virus (FV) vectors are a promising gene delivery system for use in hematopoietic stem cell gene therapy. Previous FV vector marking studies in the NOD/SCID xenotransplantation model used umbilical cord blood (UCB)-derived SCID repopulating cells (SRCs) that were assayed 5-10 weeks posttransplantation. We now report efficient FV vector transduction (>65%) of UCB-derived primitive, long-term SRCs engrafted for 18 weeks. In addition, we evaluated gene transfer into mobilized peripheral blood (MPB)-derived SRCs by improved, deleted FV vectors containing minimal cis-acting sequences and packaged by split helper constructs that would be appropriate for use in clinical trials. When used at a multiplicity of infection of 1 in a 10-hr transduction protocol, these improved vectors transduced 34% of engrafted MPB-derived SRCs.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Animals
  • Bone Marrow Cells / metabolism*
  • Bone Marrow Transplantation
  • Genetic Therapy
  • Genetic Vectors*
  • Humans
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Spumavirus*
  • Transduction, Genetic*