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Prenatal identification of potential donors for umbilical cord blood transplantation for Fanconi anemia
Abstract
Reported here are studies of Fanconi anemia fetal cells that led to the first use of umbilical cord blood for hematopoietic reconstitution in a clinical trial. Prenatal diagnosis and HLA typing were performed in fetuses at risk for Fanconi anemia (FA) to identify, prior to birth, those that were unaffected with the syndrome and were HLA‐identical to affected siblings. Umbilical cord blood was harvested at the delivery of these infants; assays of progenitor cells indicated the presence of colony‐forming units‐granulocyte‐macrophage (CFU‐GM) in numbers similar to those of bone marrow CFU‐GM that are associated with successful engraftment in HLA‐matched allogeneic bone marrow transplantation. The possibility that umbilical cord blood from a single individual can be used as an alternative to bone marrow for hematopoietic reconstitution has now been demonstrated by the successful engraftment of two patients with FA. Progenitor cell assays of umbilical cord blood collected at the birth of a child affected with FA, who had been misdiagnosed on the basis of chorionic villus sampling (CVS) studies, indicated a profound deficiency in colony formation, consistent with previously reported abnormalities in the growth of FA cells in vitro. These results suggest that the hematopoietic disorder in FA is related to an underlying problem with cell proliferation.
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“FA HSCs appear to be reduced in number early in life, even prior to the onset of overt marrow failure[35,70].”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [70]
In the citing paper
“Increasing evidence demonstrates that the bone marrow HSC/P content is reduced in FA patients, even prior to the onset of overt bone marrow failure.”
From Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia (2009) · cites this paper as [70]
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Cited by
Stem Cell Collection and Gene Transfer in Fanconi Anemia
Kelly, P. F., Radtke, S., Kalle, C. V., Balcik, B., Bohn, K., Mueller, R., Schuesler, T., Haren, M., Reeves, L., Cancelas, J. A., et al. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300033 · Cited by 180
Self-citationFinding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia
Müller, L. U. W., & Williams, D. A. (2009) · Mutation Research - Fundamental and Molecular Mechanisms of Mutagenesis · doi:10.1016/j.mrfmmm.2009.03.010 · Cited by 31
Metadata
- Authors
- Auerbach, A. D., Liu, Q., Ghosh, R., Pollack, M. S., Douglas, G. W., & Broxmeyer, H. E.
- Venue
- Transfusion
- Publisher
- Wiley
- Published
- 1990-10
- Volume
- 30
- Issue
- 8
- Pages
- 682-687
- ISSN
- 0041-1132
- 1537-2995
- Cited by
- 63
- References
- 0
- Language
- en
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