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Long-term in vivo expression of a murine adenosine deaminase gene in rhesus
monkey hematopoietic cells of multiple lineages after retroviral mediated
gene transfer into CD34+ bone marrow cells
DM Bodine, T Moritz, RE Donahue, BD Luskey, SW Kessler, DI Martin, SH Orkin, AW Nienhuis and DA Williams
Clinical Hematology Branch, National Heart, Lung, Blood Institute,
Bethesda, MD 20892.
Retroviral mediated gene transfer into stem cells has been proposed as
therapy for many inherited hematopoietic diseases. Deficiency of the enzyme
adenosine deaminase (ADA) results in depletion of T lymphocytes, causing
severe combined immunodeficiency syndrome (SCIDS). In this report, we
describe retroviral mediated gene transfer of a murine ADA cDNA into Rhesus
monkey hematopoietic stem cells. Immunoselected CD34+ bone marrow cells
were exposed to medium containing the ADA retrovirus during culture on a
stromal cell line engineered to express the transmembrane form of stem cell
factor. After infusion of autologous, transduced cells into irradiated
recipients, gene transfer was observed in all three monkeys. The ADA
provirus was detected in 2% of circulating granulocytes and T cells from
100 days post-transplantation to longer than 1 year and in B cells from 250
days post-transplantation and beyond. Mouse ADA activity was detected in
peripheral blood cells at approximately 3% the activity of monkey ADA.
Thus, we have shown gene transfer into repopulating cells that contribute
to all hematopoietic lineages with persistent gene expression. These data
provide support for the use of stem cell targeted gene transfer for therapy
of ADA deficiency.
Volume 82,
Issue 7,
pp. 1975-1980,
10/01/1993
Copyright © 1993 by The American Society of Hematology
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