Cure of murine thalassemia by bone marrow transplantation without eradication of endogenous stem cells
article
α-Thalassemic heterozygous (Hbath/+) mice were used to investigate the possible selective advantage of transplanted normal (+/+) hemopoietic cells. Without conditioning by total-body irradiation (TBI), infusion of large numbers of normal bone marrow cells failed to correct the thalassemic peripheral blood phenotype. Since the recipient's stem cells are normal with respect to number and differentiation capacity, it was thought that the transplanted stem cells were not able to lodge, or that they were not stimulated to proliferate. Therefore, a nonlethal dose of TBI was given to temporarily reduce endogenous stem cell numbers and hemopoiesis. TBI doses of 2 or 3 Gy followed by infusion of normal bone marrow cells proved to be effective in replacing the thalassemic red cells by normal red cells, whereas a dose of 1 Gy was ineffective. It is concluded that cure of thalassemia by bone marrow transplantation does not necesarily require eradication of thalassemic stem cells. Consequently, the objectives of conditioning regimens for bone marrow transplantation of thalassemic patients (and possibly other nonmalignant hemopoietic disorders) should be reconsidered. Copyright © Williams & Wilkins 1986. All Rights Reserved.
Topics
animal experimentblood and hemopoietic systembone marrow transplantationmousenonhumanpriority journalstem cellthalassemiatherapywhole body radiationAnimalBone Marrow TransplantationFemaleMaleMiceMice, Mutant StrainsPreoperative CareRadiation ChimeraRadiation DosageSupport, Non-U.S. Gov'tThalassemiaWhole-Body Irradiation
TNO Identifier
230032
ISSN
00411337
Source
Transplantation, 42(3), pp. 248-251.
Pages
248-251
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