Proceedings of the XVIITH World Congress of the Transplantation SocietyStem cell transplants in utero for genetic diseases: treatment and a model for induction of immunologic tolerance
Section snippets
Case reports
The first patient treated by in utero FLCT, in 1988, had bare lymphocyte syndrome (BLS), a genetically transmitted form of combined immunodeficiency due to the lack of HLA antigens. Infections are responsible for death in these infants, unless they grow up isolated in a fully sterile atmosphere while they are successfully reconstituted with stem cell transplants. When bone marrow transplants or FLCTs are carried out postnatally, they usually are not successful; either graft failure results from
Tolerance and MHC restriction in human chimeras
Both in patients treated by postnatal FLCT and in fetuses receiving FLCT in utero, the full reconstitution could develop despite a complete mismatch of HLA class I and class II antigens between donor and host. The separation of T- and B-lymphocyte populations, and the development of cell lines and clones from peripheral blood lymphocytes of these patients, followed by HLA typing, showed that all T lymphocytes were of donor origin, while most B lymphocytes and monocytes were of host origin in
Acknowledgements
We thank Dr. M.G. Roncarolo and Dr. H. Plotnicky for their large contribution to the studies on T-cell recognition.
References (9)
- et al.
Lancet
(1989) - et al.
Lancet
(1996) Immunol Rev
(1983)Acta Hematologica
(1996)
Cited by (12)
Donor cell replacement in mice transplanted in utero is limited by immune-independent mechanisms
2003, Blood Cells, Molecules, and DiseasesHematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival
2002, BloodCitation Excerpt :The ability to diagnose SCID in the first trimester of pregnancy led to attempts to treat this syndrome in utero with the hope that the infant would then be born with intact immunity. There are 4 literature reports of successful in utero bone marrow transplantation for SCID.18-24 These reports suggest that, although lymphoid reconstitution of SCID can be achieved with prenatal stem cell transplantation, T-cell development was not complete at birth.
In utero induction of transplantation tolerance
2001, Transplantation ProceedingsForty Years of Publication of Transplantation Proceedings—The Fourth Decade: Globalization of the Enterprise
2011, Transplantation ProceedingsCitation Excerpt :Extending this approach to achieve extended survival of DLA-identical and haploidentical grafts in dogs, Lothrop and colleagues409 used a nonmyelosuppressive regimen with transient immunosuppression using CsA plus MMF. Touraine410,411 reported that stem cell transplants delivered in utero into experimental animals achieved chimerism as well as immunomodulation. They suggested that this strategy might serve as a therapy for genetic diseases and possibly induce transplantation tolerance in humans.
Maternal and fetal safety outcomes after in utero stem cell injection: A systematic review
2023, Prenatal Diagnosis