Engraftment of gene–modified umbilical cord blood cells in neonates with adenosine deaminase deficiency (original) (raw)

References

1. Giblett, E.R., Anderson, J.E., Cohen, F., Pollara, B. & Meuwissen, H.J. Adenosine-deaminase deficiency in two patients with severely impaired cellular immunity. Lancet 2, 1067–1069 (1972).
   Article CAS PubMed Google Scholar
2. Hershfield, M.S. & Mitchell, B.S. Immunodeficiency diseases caused by adenosine deaminase deficiency and purine nucleoside phosphorylase deficiency. in The Metabolic and Molecular Bases of Inherited Disease (eds Scriver, C.R., Beaudet, A.L., Sly, W.S. & Valle, D.) 1725–1768 (McGraw-Hill, New York, 1994).
   Google Scholar
3. Parkman, R., Gelfand, E.W., Rosen, F.S., Sanderson, A. & Hirschhorn, R. Severe combined immunodeficiency and adenosine deaminase. New Engl. J. Med. 292, 714–719 (1975).
   Article CAS PubMed Google Scholar
4. Weinberg, K.I. & Kohn, D.B. Somatic gene therapy for severe combined immunodeficiency (SCID). in Somatic Gene Therapy (ed. Chang, P.) 31–47 (CRC Press, Boca Raton, 1995).
   Google Scholar
5. Blaese, R.M. Development of gene therapy for immunodeficiency: adenosine deaminase deficiency. Pediatr. Res. 33 (suppl.), S49–S55 (1993).
   Article CAS PubMed Google Scholar
6. Hershfield, M.S., Chaffee, S. & Sorensen, R.U. Enzyme replacement therapy with PEG-ADA in adenosine deaminase deficiency: Overview and case report of three patients, including two now receiving gene therapy. Pediatr. Res. 33 (suppl.), S42–S48 (1993).
   Article CAS PubMed Google Scholar
7. Broxmeyer, H.E., Cooper, S., Yoder, M. & Hangoc, G. Human umbilical cord blood as a source of transplantable hematopoietic stem and progenitor cells. Curr. Topics Microbiol. Immunol. 177, 195–204 (1992).
   CAS Google Scholar
8. Wagner, J.E. et al. Transplantation of umbilical cord blood after myeloablative therapy: Analysis of engraftment. Blood 79, 1874–1881 (1992).
   CAS PubMed Google Scholar
9. Hock, R.A., Miller, A.D. & Osborne, W.R. Expression of human adenosine deaminase from various strong promoters after gene transfer into human hematopoietic cell lines. Blood 74, 876–881 (1989).
   CAS PubMed Google Scholar
10. Williams, D.A., Lemischka, I.R., Nathan, D.G. & Mulligan, R.C. Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse. Nature 310, 476–480 (1984).
    Article CAS PubMed Google Scholar
11. Jordan, C.T. & Lemischka, I.R. Clonal and systemic analysis of long-term hematopoiesis in the mouse. Genes Dev. 4, 220–232 (1990).
    Article CAS PubMed Google Scholar
12. Triglia, T., Peterson, M.G. & Kemp, D.J. A procedure for in vitro amplification of DNA segments that lie outside the boundaries of known sequences. Nucleic Adds Res. 16, 8186 (1988).
    Article CAS Google Scholar
13. Ochman, H., Gerber, A.S. & Hartl, D.L. Genetic applications of an inverse polymerase chain reaction. Genetics 120, 621–623 (1988).
    CAS PubMed PubMed Central Google Scholar
14. Rill, D.R. et al. Direct demonstration that autologous bone marrow transplantation for solid tumors can return a multiplicity of tumorigenic cells. Blood 84, 380–383 (1994).
    CAS PubMed Google Scholar
15. Harrison, D.E. Competitive repopulation in unirradiated normal recipients. Blood 81, 2473–2474 (1993).
    CAS PubMed Google Scholar
16. Stewart, F.M., Crittenden, R.B., Lowry, P.A., Pearson-White, S. & Quesenberry, P.J. Long-term engraftment of normal and post-5-fluorouracil murine marrow into normal nonmyeloablated mice. Blood 81, 2566–2571 (1993).
    CAS PubMed Google Scholar
17. Wu, D.D. & Keating, A. Hematopoietic stem cells engraft in untreated transplant recipients. Expl. Hematol. 21, 251–256 (1993).
    CAS Google Scholar
18. Van Beusechem, V.W., Kakler, A., Meidt, P.J. & Valerio, D. Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone marrow cells. Proc. natn. Acad. Sci. U.S.A. 79, 7640–7644 (1992).
    Article Google Scholar
19. Bodine, D.M. et al. Long-term in vivo expression of a murine adenosine deaminase gene in rhesus monkey hematopoietic cells of multiple lineages after retroviral mediated gene transfer into CD34+ bone marrow cells. Blood 82, 1975–1980 (1993).
    CAS PubMed Google Scholar
20. Brenner, M.K. et al. Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients. Lancet 342, 1134–1137(1993).
    Article CAS PubMed Google Scholar
21. Valera, A., Perales, J.C., Hatzoglou, M. & Bosch, F. Expression of the neomycin-resistance (neo) gene induces alterations in gene expression and metabolism. Hum. Gene Ther. 5, 449–456 (1994).
    Article CAS PubMed Google Scholar
22. Von Melchner, H. & Housman, D.E. The expression of neomycin phospho-transferase in human promyelocytic leukemia cells (HL-60) delays their differentiation. Oncogene 2, 137–140 (1988).
    CAS PubMed Google Scholar
23. Miller, A.D. & Buttimore, C. Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production. Molec. cell. Biol. 6, 2895–2902 (1986).
    Article CAS PubMed PubMed Central Google Scholar
24. Bassin, R.H., Tuttle, N. & Fischinger, P.J. Isolation of murine sarcoma virus-transformed mouse cells which are negative for leukemia virus from agar suspension cultures. Int. J. Cancer 6, 95–107 (1970).
    Article CAS PubMed Google Scholar
25. Hanley, M.E., Nolta, J.A., Parkman, R. & Kohn, D.B. Umbilical cord blood cell transduction by retroviral vectors: Pre-clinical studies to optimize gene transfer. Blood Cells 20, 539–546 (1994).
    CAS PubMed Google Scholar
26. Hershfield, M.S. et al. Treatment of adenosine deaminase deficiency with polyethylene glycol-modifled adenosine deaminase. New Engl. J. Med. 316, 589–596 (1987).
    Article CAS PubMed Google Scholar
27. Nolta, J.A. & Kohn, D.B. Comparison of the effects of growth factors on retroviral vector-mediated gene transfer and the proliferative status of human hematopoietic progenitor cells. Hum. Gene Ther. 1, 257–268 (1990).
    Article CAS PubMed Google Scholar
28. Nolta, J.A., Yu, X.J., Bahner, I. & Kohn, D.B. Retroviral mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow. J. clin. Invest. 90, 343–348 (1992).
    Article Google Scholar
29. Weinberg, K. & Parkman, R. Severe combined immunodeficiency due to a specific defect in the production of interleukin-2. New Engl. J. Med. 322, 1718–1723 (1990).
    Article CAS PubMed Google Scholar
30. Chomczynski, P. & Sacchi, N. Single-step method of RNA isolation by acid guanidinium thiocyante-phenol-chloroform extraction. Anal. Biochem. 162, 156–159 (1987).
    Article CAS PubMed Google Scholar
31. Arredondo-Vega, F.X. et al. Paradoxical expression of adenosine deaminase in T cell cultured from a patient with adenosine deaminase deficiency and combined immunodeficiency. J. clin. Invest. 86, 444–452 (1990).
    Article CAS PubMed PubMed Central Google Scholar

Download references