In utero transplantation for thalassemia

Annals of the New York Academy of Sciences

Volumn 850, Issue , 1998, Pages 300-311

  Flake, Alan W ^a   Zanjani, Esmail D ^b  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b UNIVERSITY OF NEVADA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BONE MARROW TRANSPLANTATION; CONFERENCE PAPER; ERYTHROPOIESIS; FETUS SURGERY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNOLOGICAL TOLERANCE; THALASSEMIA;

EID: 0031852319     PISSN: 00778923     EISSN: None     Source Type: Book Series    
DOI: 10.1111/j.1749-6632.1998.tb10487.x     Document Type: Conference Paper

References (56)

1. METCALF, D. & M. A. S. MOORE. 1971. In Embryonic Aspects of Haemopoiesis. A. Neuberger & E. L. Tatum, Eds: Chapter 4. North-Holland Publishing Co. Amsterdam.
2. TAVIAN, M., L. COULOMBEL, D. LUTON, H. SAN CLEMENTE, F. DIETERLEN-LIEVRE & B. PEAULT. 1996. Aorta-associated CD34+ hematopoietic cells in the early human embryo. Blood 87: 67-72.
3. BILLINGHAM, R., L. BRENT & P. B. MEDAWAR. 1953. Actively acquired tolerance of foreign cells. Nature 172: 603-607.
4. SPRENT, J. 1995. Central tolerance of T cells. Int. Rev. Immunol. 13: 95-105.
5. ANDERSON, G., N. MOORE, J. OWEN & E. JENKINSON. 1996. Cellular interactions in thymocyte development. Annu. Rev. Immunol. 14: 73-99.
6. STROMLNGER, J. L. 1989. Developmental biology of T cell receptors. Science 244: 943-949.
7. OWEN, R. D. 1945. Immunogenetic consequences of vascular anastomoses between bovine cattle twins. Science 102: 400-401.
8. PICUS, J., W. ALDRICH & N. LETVIN. 1985. A naturally occurring bone-marrow chimeric primate. Transplantation 39: 297-303.
9. VAN DIJK, B., D. BOMMSMA & A. DE MAN. 1996. Blood group chimerism in human multiple births is not rare. Am. J. Med. Genet. 61: 264-268.
10. ANDERSON, D., R. BILLINGHAM, G. LAMPKIN & P. MEDAWAR. 1951. The use of skin grafting to distinguish between monozygotic and dizygotic twins in cattle. Heredity 5: 379-397.
11. SIMONSEN, M. 1955. The acquired immunity concept in kidney homotransplantation. Ann. N.Y. Acad. Sci. 59: 448-452.
12. FLEISCHMAN, R. & B. MINTZ. 1979. Prevention of genetic anemias in mice by microinjection of normal hematopoietic cells into the fetal placenta. Proc. Natl. Acad. Sci. USA 76: 5736-5740.
13. BLAZER, B., P. TAYLOR & D. VALLERA. 1995. In utero transfer of adult bone marrow cells into recipients with severe combined immunodeficiency disorder yields lymphoid progeny with T- and B-cell functional capabilities. Blood 86: 4353-4366.
14. ZANJANI, E. D., J. L. ASCENSAO, A. W. FLAKE, M. R. HARRISON & M. TAVASSOLI. 1992. The fetus as an optimal donor and recipient of hemopoietic stem cells. Bone Marrow Transplant. 10: 107-114.
15. FLAKE, A. W., M. R. HARRISON, N. S. ADZICK & E. D. ZANJANI. 1986. Transplantation of fetal hematopoietic stem cells in utero: the creation of hematopoietic chimeras. Science 233: 776-778.
16. FLAKE, A. & E. ZANJANI. 1997. Cellular therapy. New trends and controversies in fetal diagnosis and therapy. Obstet. Gynecol. Clin. N. Am. 24: 159-177.
17. ZANJANI, E. D., J. L. ASCENSAO & M. TAVASSOLI. 1992. Homing of liver-derived hemopoietic stem cells to fetal bone marrow. Trans. Assoc. Am. Physicians. 105: 7-14.
18. CROMBLEHOLME, T. M., M. R. HARRISON & E. D. ZANJANI. 1990. In utero transplantation of hematopoietic stem cells in sheep: the role of T cells in engraftment and graft-versus-host disease. J. Pediatr. Surg. 25: 885-892.
19. HARRISON, M. R., R. N. SLOTNICK, T. M. CROMBLEHOLME, M. S. GOLBUS et al. 1989. In-utero transplantation of fetal liver haemopoietic stem cells in monkeys. Lancet 2: 1425-1427.
20. PEARCE, R., D. KIEHM, D. ARMSTRONG, P. LITTLE et al. 1989. Induction of hematopoietic chimerism in the caprine fetus by intraperitoneal injection of fetal liver cells. Experientia 45: 307-308.
21. CARRIER, E., T. LEE, M. BUSCH & M. COWAN. 1995. Induction of tolerance in nondefective mice after in utero transplantation of major histocompatibility complex-mismatched fetal hematopoietic stem cells. Blood. 86: 4681-4690.
22. ZANJANI, E. D., M. G. PALLAVICINI, J. L. ASCENSAO, A. W. FLAKE et al. 1992. Engraftment and long-term expression of human fetal hemopoietic stem cells in sheep following transplantation in utero. J. Clin. Invest. 89: 1178-1188.
23. SHIELDS, L., E. BRYANT, T. EASTERLING & R. ANDREWS. 1995. fetal liver cell transplantation for the creation of lymphhematopoietic chimerism in the fetal baboon. Am. J. Obstet. Gynecol. 173: 1157-1160.
24. PALLAVICINI, M. G., A. W. FLAKE, D. MADDEN, C. BETHEL et al. 1992. Hemopoietic chimerism in rodents transplanted in utero with fetal human hemopoietic cells. Transplant. Proc. 24: 542-543.
25. HEDRICK, M. H., H. E. RICE, D. H. SACHS, E. D. ZANJANI & A. W. FLAKE. 1993. Creation of pig-sheep xenogeneic hematopoietic chimerism by the in utero transplantation of hematopoietic stem cells. Transplant. Sci. 3: 23-26.
26. RICE, H. E., M. H. HEDRICK & A. W. FLAKE. 1994. In utero transplantation of rat hematopoietic stem cells induces xenogeneic chimerism in mice. Transplant. Proc. 26: 126-128.
27. ZANJANI, E. D., A. W. FLAKE, H. RICE, M. HEDRICK & M. TAVASSOLI. 1994. Long-term repopulating ability of xenogeneic transplanted human fetal liver hematopoietic stem cells in sheep. J. Clin. Invest. 93: 1051-1055.
28. KAWASHLMA, I., E. D. ZANJANI, G. ALMAIDA-PORADA, A. W. FLAKE et al. 1996. CD34+ human marrow cells that express low levels of Kit protein are enriched for long-term marrow-engrafting cells. Blood 87: 4136-4142.
29. CIVIN, C., G. ALMEIDA-PORADA, M.-J. LEE, J. OLWEUS et al. 1996. Sustained, retransplantable, multilineage engraftment of highly purified adult human bone marrow stem cells in vivo. Blood 88: 4102-4109.
30. ZANJANI, E. D., A. W. FLAKE, H. E. RICE, M. H. HEDRICK & M. TAVASSOLI. 1993. An in vivo comparison of potential human donor hematopoietic stem cell sources for bone marrow transplantation using the human/sheep xenograft model. Blood 82 (Suppl. 1): 655a (Abstract 2605).
31. SROUR, E. F., E. D. ZANJANI, K. CORNETTA, C. M. TRAYCOFF et al. 1993. Persistence of human multilineage, self-renewing lymphohematopoietic stem cells in chimeric sheep. Blood 82: 3333-3342.
32. TRAYCOFF, C. M., R. HUFFMAN, E. D. ZANJANI, K. CORNETTA et al. 1994. Measurement of marrow repopulating potential of human hematopoietic progenitor and stem cells using a fetal sheep model. Prog. Clin. Biol. Res. 389: 281-291.
33. UCHIDA, N., J. COMBS, S. CHEN, E. ZANJANI et al. 1996. Primitive human hematopoietic cells displaying differential efflux of the rhodamine 123 dye have distinct biological activities. Blood 88: 1297-1305.
34. FLAKE, A. & E. ZANJANI. 1998. In utero hematopoietic stem cell transplantation: A status report. J. Am. Med. Assoc. 278: 932-937.
35. LINCH, D., C. RODECK & K. NICOLAIDES. 1986. Attempted bone marrow transplantation in a 17 week fetus. Lancet 1: 1382.
36. TOURALVE, J. L. 1991. Stem cell transplantation in primary immunodeficiency, with special reference to the first prenatal, in utero, transplants. Allergol. Immunopathol. (Madr) 19: 49-51.
37. TOURALNE, J. L., D. RAUDRANT, C. ROYO, A. REBAUD et al. 1989. In-utero transplantation of stem cells in bare lymphocyte syndrome. Lancet 1: 1382.
38. TOURAINE, J. 1996. Treatment of human fetuses and induction of immunological tolerance in humans by in utero transplantation of stem cells into fetal recipients. Acta Haematol. 96: 115-119.
39. WENGLER, G., A. LANFRANCHI, T. FRUSCA, R. VERARDI et al. 1996. In-utero transplantation of parental CD34 haematopoietic progenitor cells in a patient with X-linked severe combined immunodeficiency (SCIDX1). Lancet 348: 1484-1487.
40. FLAKE, A., M.-G. RONCAROLO, J. PUCK, G. ALMEIDA-PORADA et al. 1996. Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow. N. Engl. J. Med. 335: 1806-1810.
41. COWAN, M. & M. GOLBUS. 1994. In utero hematopoietic stem cell transplants for inherited disease. Am. J. Pediatr. Hemat./Oncol. 16: 35-42.
42. WESTGREN, M., O. RINGDEN, E.-N. STURLA, E. SVERKER et al. 1996. Lack of evidence of permanent engraftment after in utero fetal stem cell transplantation in congenital hemoglobinopathies. Transplantation 61: 1176-1179.
43. GUPTA, S., R. PAHWA, R. O'REILLY, R. GOOD & F. SIEGEL. 1976. Ontogeny of lymphocyte subpopulations in human fetal liver. Proc. Natl. Acad. Sci. USA 73: 919-922.
44. PAHWA, R., S. PAHWA, R. GOOD, G. INCEFY & R. O'REILLY. 1977. Rationale for combined use of fetal liver and thymic for immunological reconstitution in patients with severe combined immunodeficiency. Proc. Natl. Acad. Sci. USA 74: 3002-3005.
45. HAYWARD, A., J. HOBBINS, R. QUINONES, R. GILLER et al. 1996. Michrochimerism and tolerance following intrauterine transplantation and transfusion for a-thalassemia-1. Presented at the In Utero Stem Cell Transplantation and Gene Therapy Conference. Reno, NV, 1996.
46. BUNN, H. & B. FORGET. 1986. In Hemoglobin Structure. Chapter 2: 13-35. W. B. Saunders. Philadelphia.
47. LAM, Y., A. GHOSH, M. TANG, C. LEE & S. SIN. 1997. Second-trimester hydrops fetalis in pregnancies affected by homozygous α thalassemia-1. Prenat. Diagn. 17: 267-269.
48. BETHEL, C., D. MURUGESH, M. HARRISON, N. MOHANDAS & E. RUBIN. 1993. Selective erythroid replacement in murine b-thalassemia using fetal hematopoietic stem cells. Proc. Natl. Acad. Sci. USA 90: 10120-10124.
49. VAN DEN BOS, C., D. KIEBOOM, J. VAN DER SLUIJS, M. BAERT et al. 1994. Selective advantage of normal erythrocyte production after bone marrow transplantation of a-thalassemic mice. Exp. Hematol. 22: 441-446.
50. ANDREANI, M., M. MANNA, G. LUCARELLI, P. TONUCCI et al. 1996. Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood 87: 3494-3499.
51. KAPELUSHNIK, J., R. OR, D. FILON, A. NAGLER et al. 1995. Analysis of β-globin mutations shows stable mixed chimerism in patients with thalassemia after bone marrow transplantation. Blood 86: 3241-3246.
52. KAUFMAN, C. L. & S. T. ILDSTAD. 1994. Induction of donor-specific tolerance by transplantation of bone marrow. Therap. Immunol. 1: 101-111.
53. SYKES, M. 1996. Immunobiology of transplantation. FASEB J. 10: 721-730.
54. OR, R., J. KAPELUSHNIK, E. NAPARSTEK, A. NAGLER et al. 1996. Second transplantation using allogeneic peripheral blood stem cells in a β-thalassaemia major patient featuring stable mixed chimaerism. Br. J. Haematol. 94: 285-287.
55. FLETCHER, J. C. 1992. fetal therapy, ethics and public policies, fetal Diagn Ther. 7: 158-168.
56. SLAVIN, S., E. NAPARSTEK & M. ZIEGLER. 1992. Clinical application of intrauterine bone marrow transplantation for treatment of genetic disease-feasibility studies. Bone Marrow Transplant. 1: 189-190.