Long-term in vivo expression of the MFG-ADA retroviral vector in rhesus monkeys transplanted with transduced bone marrow cells

L C Kaptein, V W Van Beusechem, I Rivière, R C Mulligan, D Valerio

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We have tested the recombinant human adenosine deaminase (hADA) retroviral vector MFG-ADA for its efficacy in transducing hemopoietic stem cells of nonhuman primates and its expression level in the hematopoietic system. The percentage of provirus-positive granulocytes 1 year after transplantation of bone marrow transduced with MFG-ADA was 0.1%, which was equivalent to previously obtained results with the hADA virus-producing cell line POC-1. However, in MFG-ADA monkeys, significantly more peripheral blood mononuclear cells carried the hADA gene (1% versus 0.1%). Human ADA expression levels in peripheral blood mononuclear cells were different between POC-1 and MFG-ADA monkeys using samples with equal numbers of provirus copies per cell. In contrast, in total red blood cell lysates of MFG-ADA monkeys, the hADA expression was higher (approximately 10-fold) and could be detected longer (20 weeks and up to more than 1 year after bone marrow transplantation in 2 monkeys) than in POC-1 monkeys that were only positive for up to 12 weeks at the most. At 3 years after bone marrow transplantation, the MFG-ADA provirus could still be detected in 0.1% of bone marrow cells and peripheral blood cells and in 1% of cultured T cells. These results show that MFG-ADA virus can give rise to long-term in vivo expression of hADA in the primate hematopoietic system. However, transduction efficiencies remain low.

Original languageEnglish
Pages (from-to)1605-10
Number of pages6
JournalHuman gene therapy
Issue number13
Publication statusPublished - 1 Sept 1997


  • 3T3 Cells
  • Adenosine Deaminase/genetics
  • Animals
  • Bone Marrow Transplantation
  • Coculture Techniques
  • Genetic Vectors
  • Hematopoietic Stem Cells/enzymology
  • Humans
  • Lymphocyte Depletion
  • Macaca mulatta
  • Mice
  • Transduction, Genetic

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