Abstract
Hemopoietic progenitor cells have been considered an ideal target for gene therapy of many single gene disorders currently treated by bone marrow transplantation as they are easily obtained and manipulated ex vivo. Furthermore genetic modification of stem cells could be sufficient to repopulate the hemopoietic and lymphoid system of an individual for their entire life. However, the level of gene transfer obtained with current gene transfer techniques as wells as concerns about the expression and regulation of new genes in such cells has delayed the use of gene therapy in most patients with these conditions (Miller, 1992; Anderson, 1992). In contrast gene marking studies allow clinically biological questions to be addressed even with the limited efficiency of gene transfer available with current vectors. In our initial studies in autologous bone marrow transplantation (AMBT), we used gene marking techniques to determine the source of relapse after ABMT and to learn more about the biology of normal marrow reconstitution. In follow up studies we are evaluating the efficacy of purging and the effect of growth factors on reconstitution. We are also using gene marking to monitor the efficacy of an adoptive transfer approach in patients at high risk of developing EBV lymphoproliferation following allogeneic bone marrow transplantation.
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© 1996 Springer-Verlag Berlin Heidelberg
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Heslop, H.E., Rooney, C.M., Rill, D.R., Brenner, M.K. (1996). Gene Transfer into Hemopoietic Progenitors: Implications for Bone Marrow Transplantation. In: Zander, A.R., Ostertag, W., Afanasiev, B.V., Grosveld, F. (eds) Gene Technology. NATO ASI Series, vol 94. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-61122-3_14
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DOI: https://doi.org/10.1007/978-3-642-61122-3_14
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