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Autologous Bone Marrow Transplantation - Blog Science Connections

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Expression of Human Adenosine Deaminase in<br />

the Hematopoietic Cells of Monkeys<br />

Martin A. Eglitis, Philip W. Kantoff, Al Gillio, Claudio Boridignon,<br />

Jeanne R. McLachlin, Robert C. Moen, Nancy A. Kernan,<br />

Donald B. Kohn, Sheau-Fung Yu, Stefan Karlsson,<br />

Euelyn M. Karson, James A. Zwiebel, Eli Gilboa,<br />

R. Michael Blaese, Arthur Nienhuis,<br />

Richard J. O'Reilly, and<br />

W. French Anderson<br />

Recently developed retroviral vectors appear to be promising as vehicles for<br />

introducing genes into bone marrow cells. A number of qualities make them<br />

particularly attractive for gene transfer: 1) they have a wide host range, 2) a<br />

single copy can be inserted into the genomic DNA of the cell, and 3) they can<br />

be inserted with no apparent harm to the cell. Several laboratories have used<br />

such vectors successfully to transfer functioning genes into the hematopoietic<br />

cells of mice, both in vitro and in vivo (1-5).<br />

Based on such early successes in mice, we have attempted to establish a<br />

model for autologous bone marrow transplant/gene transfer in nonhuman<br />

primates. These studies have been carried out in two locations with two different<br />

species of monkey. One was at the Memorial Sloan-Kettering Cancer<br />

Center using cynomolgus macaques, which have been used there for several<br />

709

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