Abstract
The possibility that human genetic diseases may be treated by transfer of normal genes to correct abnormalities caused by defective genes is becoming more practical with recent advances in techniques for efficient gene transfer (1). The unique structure and mode of propagation of retroviruses make them well-suited for gene transfer. Bone marrow is a likely initial target for gene therapy, because extensive experience exists in manipulating and treating this tissue ex vivo and because some genetic diseases exert a primary tissue effect in the marrow. Several groups have reported successful transfer and expression of exogenous genes in hematopoietic progenitor cells (2–24). Most in vivo studies have focused on mice using vectors derived from the Moloney murine leukemia virus (11,14,15,17,18,20). The expression of retroviral vector transduced genes in hematopoietic stem cells in vivo has been variable in most species but is generally transient and only involves a small fraction of the hematopoietic cells (11,14,15,18,20–24). Stable long term expression of retroviral transduced genes in hematopoietic progenitor cells in species other than mice has not been reported. We found that canine and feline hematopoietic progenitor cells could also be infected with retroviral vectors carrying the neomycin phosphotransferase (neoR) gene (2,12). Therefore, the dog and cat are excellent large animal models to test gene therapy because they have a number of genetic disorders similar to human diseases. Furthermore, the dog is an established preclinical model of bone marrow transplantation.
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Al-Lebban, Z.S., Jones, J.B., Eglitis, M.A., Anderson, W.F., Lothrop, C.D. (1989). Gene Transfer in Canine and Feline Hematopoietic Progenitor Cells with Retroviral Vectors. In: Baum, S.J., Dicke, K.A., Lotzová, E., Pluznik, D.H. (eds) Experimental Hematology Today—1988. Experimental Hematology Today—1988, vol 1988. Springer, New York, NY. https://doi.org/10.1007/978-1-4613-8862-3_16
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DOI: https://doi.org/10.1007/978-1-4613-8862-3_16
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