Abstract
Allogeneic bone marrow (BM) stem cell transplantation in childhood is used to cure a variety of inherited diseases including severe immunodeficiency diseases, hemoglobinopathies, and some storage diseases (O’Reilly et al. 1984; Cowan 1991). For all of these disorders, except most types of severe combined immunodeficiency disease (SCID), immunosuppressive chemotherapy is required to prevent graft rejection. Also, in order to obtain sufficient erythroid and/or myeloid engraftment, marrow ablative chemotherapy is necessary to “make space” for the donor hematopoietic stem cells (HSC). This conditioning is associated with significant morbidity and a mortality of 10–15%. Finally, for some diseases (e. g., Hurler’s mucopolysaccharidosis), significant organ damage has already occurred by the time the diagnosis is made postnatally (Hobbs 1988). The need for immunosuppression can be eliminated if the transplant is done sufficiently early in utero when the fetus is unable to reject allogeneic donor HSC.
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Cowan, M.J., Chou, SH., Tarantal, A.F. (2001). Tolerance Induction Post In Utero Stem Cell Transplantation. In: Holzgreve, W., Lessl, M. (eds) Stem Cells from Cord Blood, in Utero Stem Cell Development and Transplantation-Inclusive Gene Therapy. Ernst Schering Research Foundation Workshop, vol 33. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-662-04469-8_10
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DOI: https://doi.org/10.1007/978-3-662-04469-8_10
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