Increased Efficiency of Retroviral-Mediated Gene Transfer and Expression in Primate Bone Marrow Progenitors after 5-Fluorouracil-Induced Hematopoietic Suppression and Recovery
NAVAL MEDICAL RESEARCH INST BETHESDA MD
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Retroviral vecotrs have been used to transfer and express cDNAs in murine and human hematopoietic cells in vitro. DNA sequences were also introduced into murine hematopoietic progenitor cells in vivo and expression of the transferred gene was obtained after in vitro transductionsyngeneic bone marrow transplantation. Long-term in vivo expression in hematopoietic cells showed successful transduction of early, self-renewing hematopoietic progenitors. These data have raised the expectation that gene therapy may be useful in treating human genetic disease. An approved clinical trial using retroviral-mediated gene transfer is being performed in humans using the neomycin phosphotransferase NPT gene transferred into tumor-infiltrating lymphocytes as a marker gene. Gene transfer into bone marrow progenitors as a therapeutic measure awaits successful completion of primate experiments. To date, long-term retroviral vector expression in primate bone marrow progenitors using amphotropically packaged vectors has met with limited success.
- Medicine and Medical Research