Differential transduction efficiency of SCID-repopulating cells derived from umbilical cord blood and granulocyte colony-stimulating factor-mobilized peripheral blood

Hum Gene Ther. 2001 Nov 20;12(17):2095-108. doi: 10.1089/10430340152677430.

Abstract

The gene transfer efficiency into nonobese diabetic/severe combined immunodeficient (NOD/SCID)-repopulating cells (SRCs) derived from umbilical cord blood (UCB) (n = 11 NOD/SCID mice) and granulocyte-colony stimulating factor (G-CSF)-mobilized peripheral blood (MPB) (n = 64 NOD/SCID mice) was compared using a clinically relevant protocol and a retrovirus vector expressing the enhanced green fluorescent protein (EGFP). At 6-9 weeks after transplantation, the frequency of transduced human cells in the bone marrow (BM) (40.5% +/- 2.4% [mean +/- SE]) and spleen (SPL) (36.4% +/- 3.2%) in recipients of UCB cells was significantly higher (p < 0.001) than that observed in the BM (2.2% +/- 1.8%) and SPL (2.0% +/- 2.6%) in recipients of MPB. In subsequent studies, MPB was cultured for 2-8 days in cytokines prior to transduction to determine if longer prestimulation was required for optimal gene transfer. A significant increase in gene transfer into CD45(+) human cells and clonogenic cells derived from MPB SRCs was observed when cells were prestimulated for 6 days compared to 2 days prior to transduction (p = 0.019). However, even after 6 days of prestimulation, transduction was still significantly less than UCB. A substantial discrepancy exists in the ability to introduce genes effectively via retrovirus vectors into SRCs derived from MPB as compared to UCB.

Publication types

  • Comparative Study
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Animals
  • Blood Cells / cytology
  • Blood Cells / drug effects*
  • Blood Cells / metabolism*
  • Blood Cells / transplantation
  • Blood Transfusion*
  • Bone Marrow Cells / cytology
  • Bone Marrow Cells / drug effects
  • Bone Marrow Cells / metabolism
  • Bone Marrow Transplantation
  • Colony-Forming Units Assay
  • Fetal Blood / cytology
  • Fetal Blood / metabolism*
  • Flow Cytometry
  • Gene Expression
  • Genetic Therapy / methods
  • Granulocyte Colony-Stimulating Factor / pharmacology*
  • Green Fluorescent Proteins
  • Humans
  • Leukocyte Common Antigens / analysis
  • Leukocyte Common Antigens / immunology
  • Luminescent Proteins / analysis
  • Luminescent Proteins / genetics
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Polymerase Chain Reaction
  • Retroviridae / genetics
  • Severe Combined Immunodeficiency / immunology*
  • Spleen / cytology
  • Spleen / metabolism
  • Time Factors
  • Transduction, Genetic / methods*
  • Transgenes / genetics
  • Transplantation Immunology

Substances

  • Luminescent Proteins
  • Granulocyte Colony-Stimulating Factor
  • Green Fluorescent Proteins
  • Leukocyte Common Antigens