Applications of hematopoietic stem cells and gene transfer

Transfusion Medicine and Hemotherapy

Volumn 23, Issue 3, 1996, Pages 147-160

  Huss, R ^a  

^a UNIVERSITY OF MUNICH   (Germany)

Author keywords

Gene transfer; Hematopoietic stem cells; Transplantation

Indexed keywords

GENE THERAPY; GENE TRANSFER; HEMATOPOIETIC STEM CELL; HUMAN; REVIEW; STEM CELL TRANSPLANTATION;

ANIMALS; COMBINED MODALITY THERAPY; GENE TRANSFER TECHNIQUES; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MICE; TREATMENT OUTCOME;

EID: 0029681792     PISSN: 16603796     EISSN: 16603818     Source Type: Journal    
DOI: 10.1159/000223285     Document Type: Article

References (224)

1. Appelbaum FR, Barrall J, Storb R, Fisher LD, Schoch G, Ramberg RE, Shulman H, Anasetti C, Bearman SI, Beatty P, Bensinger WI, Buckner CD, Clift RA, Hansen JA, Martin P, Petersen FB, Sanders JE, Singer J, Stewart P, Sullivan KM, Witherspoon RP, Thomas ED: Bone marrow transplantation for patients with myelodysplasia, Pretreatment variables and outcome, Ann Intern Med 1990:112:590-597.
2. Armitage JO: Bone marrow transplantation in the treatment of patients with lymphoma, Blood 1989; 73:1749-1758.
3. Flowers MED, Doney KC, Storb R, Deeg HJ, Sanders JE, Sullivan KM, Brvanl E, Witherspoon RP, Appelbaum FR, Buckner CD, Hansen JA, Thomas ED: Marrow transplantation for Fanconi anemia with or without leukemic transformation: An update of the Seattle experience, Bone Marrow Transplant 1992;9:167-173.
4. Gahrton G, Tura S, Ljungman P, Belanger B, Brandt L, Cavo M, Façon T, Granena A, Gratwohl A, Löwenberg B, Nikoskelainen J, Reiffers J, Samson D, Selby P, Volin L, for the European Group for Bone Marrow Transplantation: Allogeneic bone marrow transplantation in multiple myeloma, N Engl J Med 1991:325:1267-1273.
5. Lucarelli G, Galimberti M, Polchi P, Angelucci E, Baronciani D, Giardini C, Politi P, Durazzi SMT, Muretto P, Albertini F: Bone marrow transplantation in patients with thalassemia, N Engl J Med 1990: 322:417-421.
6. Reece DE, Barnett MJ, Connors JM, Fairev RN, Greer JP, Herzig GP, Herzig RH, Klingemann H-G, O’Reilly SE, Shepherd JD, Spinelli JJ, Voss NJ, Wolff SN, Phillips GL: Intensive chemotherapy with cyclophosphamide, carmustinc, and etoposide followed by autologous bone marrow transplantation for relapsed Hodgkin’s disease, J Clin Oncol 1991:9: 1871-1879.
7. Santos GW: Marrow transplantation in acute non lymphocytic leukemia, Blood 1989:74:901-908.
8. Storb R, Anasetti C, Appelbaum F, Bensinger W, Buckner CD, Clift R, Deeg HJ, Doney K, Hansen J, Loughran T, Martin P, Pepe M, Petersen F, Sanders J, Singer J, Stewart P, Sullivan KM, Witherspoon R, Thomas ED: Marrow transplantation for severe aplastic anemia and thalassemia major, Semin Hematol 1991:28:235-239.
9. Thomas ED, Clift RA, Fefer A, Appelbaum FR, Beatty P, Bensinger WI, Buckner CD, Cheevor MA, Deeg HJ, Doney K, Flournoy N, Greenberg P, Hansen JA, Martin P, McGuffin R, Ramberg R, Sanders JE, Singer J, Stewart P, Storb R, Sullivan K, Weiden PL, Witherspoon R: Marrow transplantation for the treatment of chronic myelogenous leukemia, Ann Intern Med 1986;104:155-163.
10. Fischer A, Durandy A, de Villartay JP, Vilmer E, Le Deist F, Gerota I, Griscelli C: HLA-haploidentical bone marrow transplantation for severe combined immunodeficiency using E rosette fractionation and cyclosporine, Blood 1986:67:444-449.
11. Krivit W, Whitley CB, Chang P-N: Lysosomal storage diseases treated by bone marrow transplantation: in Gale RP, Champlin RE (eds): Bone Marrow Transplantation: Current Controversies, Vol, 91 of UCLA Symposia on Molecular and Cellular Biology, New York, Alan R, Liss, 1989, pp 367-378.
12. Lane HC, Zunich KM, Wilson W, Cefali F, Faster M, Kovacs JA, Masur H, Lcitman SF, Klein HG, Steis RG, Iaingo DL, Fauci AS: Syngeneic bone marrow transplantation and adoptive transfer of peripheral blood lymphocytes combined with zidovudine in human immunodeficiency virus (HIV) infection, Ann Intern Med 1990:113:512-519.
13. Hughes WT, Kuhn S, C'haudhary S, Feldman S, Verosa M, Aur RJA, Pratt C, George SL: Successful chemoprophylaxis for pneumocystis carinii pneumonitis, N Engl J Med 1977:297:1419-1426.
14. Meyers JD, Wade JC, Shepp DH, Newton B: Acyclovir treatment of varicclla-zoster virus infection in the compromised host, Transplantation 1984:37: 571-574.
15. Schmidt GM, Horak DA, Niland JC, Duncan SR, Forman SJ, Zaia JA: A randomized, controlled trial of prophylactic ganciclovir for cytomegalovirus pulmonary infection in recipients of allogeneic bone marrow transplants, N Fngl J Med 1991:324: 1005-1011.
16. Demelri GD: Hematopoietic growth factors: Current knowledge, future prospects, Curr Probl Cancer 1992:16:177-259.
17. Licschkc GJ, Burgess AW: Granulocyte colony-stimulating factor and granulocyte-macrophage colony-stimulating factor (I), N Engl J Med 1992: 327:28-35.
18. Lieschke GJ, Burgess AW: Granulocyte colony-stimulating factor and granulocyte-macrophage colony-stimulating factor (2), N Engl J Med 1992: 327:99-106.
19. Nemunaitis J, Rabinowe SN, Singer JW, Bierman PJ, Vosc JM, Freedman AS, Onetto N, Gillis S, Oette D, Gold M, Buckner CD, Hansen JA, Ritz J, Appel-baum FR, Armitage JO, Nadler LM: Recombinant granulocyte-macrophage colony-stimulating factor after autologous bone marrow transplantation for lymphoid cancer, N Engl J Med 1991:324:1773-1778.
20. Nemunaitis J, Singer JW, Buckner CD, Durnam D, Epstein C, Hill R, Storb R, Thomas ED, Appelbaum FR: The use of recombinant human granulocvte-macrophagc colony-stimulating factor in graft failure after bone marrow transplantation, Blood 1990: 76:245-253.
21. Sheridan WP, Morstyn G, Wolf M, Dodds A, Lusk J, Maher D, Layton JE, Green MD, Souza L, Fox RM: Granulocyte colony-stimulating factor and neutrophil recovery after high-dose chemotherapy and autologous bone marrow transplantation, Uancet 1989:2:891-895.
22. Taylor KM, Jagannath S, Spitzer CI, Spinolo JA, Tucker SL, Fogel B, Cabanillas FF, Hagemeister FB, Souza LM: Recombinant human granulocyte colony-stimulating factor hastens granulocyte recovery after high-dose chemotherapy and autologous bone marrow transplantation in Hodgkin's disease, J Clin Oncol 1989:7:1791-1799.
23. Anasetti C, Amos D, Beatty PG, Appelbaum FR, Bensinger W, Buckner CD, Clift R, Doney K, Martin PJ, Mickelson E, Nisperos B, O'Quigley J, Ramberg R, Sanders JE, Stewart P, Storb R, Sullivan KM, Witherspoon RP, Thomas ED, Hansen JA: Effect of HLA compatibility on engraftment of bone marrow transplants in patients with leukemia or lymphoma, N Engl J Med 1989:320:197-204.
24. Beatty PG, Anasetti C, Thomas ED, Hansen JA: Marrow transplantation from relatives other than HLA-identical siblings: in Gale RP, Champlin R (eds): Bone Marrow Transplantation: Current Controversies, UCLA Symposia on Molecular and Cellular Biology, New Series, Vol, 91, New York, Alan R, Liss, Inc, 1989, pp 619-624.
25. Gingrich RD, Ginder GD, Goeken NE, Ilowe CWS, Wen BC, Hussey DM, Fyfe MA: Allogeneic marrow grafting with partially mismatched, unrelated marrow donors, Blood 1988:71:1375-1381.
26. Hansen JA, Clift RA, Thomas ED, Buckner CD, Storb R, Giblett ER: Transplantation of marrow from an unrelated donor to a patient with acute leukemia, N Engl J Med 1980:303:565-567.
27. Hows JM, Yin JL., Marsh J, Swirsky D, Jones L., Apperlev JF, James DCO, Smithers S, Batchelor JR, Goldman JM, Gordon-Smith EC: Histocompatible unrelated volunteer donors compared with HLA nonidentical family donors in marrow transplantation for aplastic anemia and leukemia, Blood 1986: 68:1322-1328.
28. Kersey JFI, Weisdorf D, Ncsbit ME, LeBien TW, Woods WG, McGlave PB, Kim T, Vallera DA, Goldman AL Bostrom B, Hurd D, Ramsay NKC: Comparison of autologous and allogeneic bone marrow transplantation for treatment of high-risk refractory acute lymphoblastic leukemia, N Engl J Med 1987: 317:461-467.
29. McGlave PB, Beatty P, Ash R, Hows JM: Therapy for chronic myelogenous leukemia with unrelated donor bone marrow transplantation: Results in 102 cases, Blood 1990:75:1728-1732.
30. Deeg HJ, Cottler-Fox M: Clinical spectrum and pathophysiology of acute graft-vs.-host disease: in Burakoff SJ, Deeg HJ, Ferrara J, Atkinson K (eds): Graft-vs.-Host Disease: Immunology, Pathophysiology, and Treatment, New York, Marcel Dekker, Inc., 1990, pp 311-335.
31. Hood AF, Vogelsang GB, Black LP, Farmer ER, Santos GW: Acute graft-vs-host disease, Development following autologous and syngeneic bone marrow transplantation, Arch Dermatol 1987:123: 745-750.
32. Korngold R, Sprent J: T cell subsets in graft-vs.-host disease: in Burakoff SJ, Deeg HJ, Ferrara J, Atkinson K (eds): Graft-vs.-Host Disease: Immunology, pathophysiology, and treatment, New York, Marcel Dekker, Inc., 1990, pp 31-50.
33. Martin PJ: Donor CD8 cells prevent allogeneic marrow graft rejection in mice: Potential implications for marrow transplantation in humans, J Exp Med 1993:178:703-712.
34. Weiden PL, Sullivan KM, Flournoy N, Storb R, Thomas ED, and the Seattle Marrow Transplant Team: Antileukemic effect of chronic graft-versus-host disease, Contribution to improved survival after allogeneic marrow transplantation, N Engl J Med 1981:304:1529-1533.
35. Hughes WT, Armstrong D, Bodey GP, Feld R, Man-dell GL, Meyers JD, Pizzo PA, Schimpff SC, Shcnep JL, Wade JC, Young US, Yow MD: The Infectious Diseases Society of America: Guidelines for the use of antimicrobial agents in neutropenic patients with unexplained fever, J Infect Dis 1990:161:381-396.
36. Wingard JR: Management of infectious complications of bone marrow transplantation, Oncology (Williston Park) 1990:4:69.
37. Nienhuis AW, McDonagh KT, Bodine DM: Gene transfer into hematopoietic stem cells, Cancer 1991:67:2700-2704.
38. Wilson JM, Danos O, Grossman M, Raulet DII, Mulligan RC: Expression of human adenosine deaminase in mice reconstituted with retrovirus-transduced hematopoietic stem cells, Proc Natl Acad Sci USA 1990:87:439-443.
39. Beatty PG, Clift RA, Mickelson EM, Nisperos B, Flournoy N, Martin PJ, Sanders JE, Stewart P, Buckner CD, Storb R, Thomas ED, Hansen JA: Marrow transplantation from related donors other than HLA-identical siblings, N Engl J Med 1985: 313:765-771.
40. Sullivan KM, Witherspoon RP, Storb R, Buckner CD, Sanders J, Thomas ED: Long-term results of allogeneic hone marrow transplantation, Transplant Proc 1989;21:2926-2928.
41. Beatty PG, Dahlberg S, Mickelson EM, Nisperos B, Opelz G, Martin PJ, Hansen JA: Probability of finding H LA-matched unrelated marrow donors, Transplantation 1988:45:714-718.
42. Ash RC, Casper JT, Chitambar CR, Hansen R, Bunin N, Truitt RL, Lawton C, Murray K, Hunter J, Baxter-Lowe LA, Gottschall JL, Oldham K, Anderson T, Camitta B, Menitove J: Successful allogeneic transplantation of T-cell-depleted bone marrow from closely HLA-matched unrelated donors, N Engl J Med 1990:322:485-494.
43. de Witte T, Hoogcnhout J, de Pauw B, Holdrinet R, Janssen J, Wessels J, van Daal W, Huslinx T, Hannen C: Depletion of donor lymphocytes by counterflow centrifugation successfully prevents acute graft* versus-host disease in matched allogeneic marrow' transplantation, Blood 1986;67:1302-1308.
44. Hale G, Cobbold S, Waldmann II: T cell depletion with Campath-1 in allogeneic bone marrow transplantation, Transplantation 1988:45:753-759.
45. Marmont AM, Horowitz MM, Gale RP, Sobocinski K, Ash RC, van Bekkum DW, Champlin RE, Dicke KA, Goldman JM, Good RA, Herzig RH, Hong R, Masaoka T, Rimm AA, Ringdcn O, Speck B, Weiner RS, Bortin MM: T-cell depletion of HLA-identical transplants in leukemia, Blood 1991:78: 2120-2130.
46. Mitsuvasu RT, Champlin RE, Gale RP, Ho WG, Lenarsky C, Winston D, Selch M, Elashoff R, Giorgi JV, Wells J, Tcrasaki P, Billing R, Feig S: Treatment of donor bone marrow with monoclonal anti-T-ccll antibody and complement for the prevention of graft-versus-host disease, Ann Intern Med 1986: 105:20-26.
47. Anasetti C, Martin PJ, Storb R, Appelbaum FR, Beatty PG, Davis J, Doney K, Hill HE, Stewart P, Sullivan KM, Witherspoon RP, Thomas ED, Hansen JA: Treatment of acute graft-versus-host disease with a nonmitogenic anti-CD3 monoclonal antibody, Transplantation 1992:54:844-851.
48. Anlin JH, Bierer BE, Smith BR, Ferrara J, Guinan EC, Sicff G, Golan DE, Macklis RM, Tarbcll NJ, Lynch E, Reichert TA, Blythman H, Bouloux C, Rappcport JM, Burakoff SJ, Weinstein HJ: Selective depletion of bone marrow T lymphocytes with anti* CD5 monoclonal antibodies: Effective prophylaxis for graft-versus-host disease in patients with hematologic malignancies, Blood 1991;78:2139-2149.
49. Champlin R, Ho W, Gajewski J, Feig S, Burnison M, Holley G, Greenberg P, Lee K, Schmid I, Giorgi J, Yam P, Petz L, Winston D, Warner N, Reichert T: Selective depletion of CD8+ T lymphocytes for prevention of graft-versus-host disease after allogeneic bone marrow transplantation, Blood 1990:76: 418-423.
50. Gleixner B, Kolb HJ, Holler E, Liescnfeld S, Riedner C, Hiller E, Seeber Ch, Kempeni J, Möller A, Knabe H, Mempel W, Ledderose G, Wilmanns W: Treatment of aGVHD with OKT3: Clinical outcome and side-effects associated with release of TNFa, Bone Marrow Transplant 1991:8:93-98.
51. Gratama JW, Jansen J, Lipovich RA, Tanke HJ, Goldstein G, Zwaan FE: Treatment of acute graft-versus-host disease with monoclonal antibody OKT3, Clinical results and effect on circulating T lymphocytes, Transplantation 1984:38:469-474.
52. Martin PJ, Hansen JA, Anasetti C, Zutter M, Durnam D, Storb R, Thomas ED: Treatment of acute graft-versus-host disease with anti-CD3 monoclonal antibodies, Am J Kidney Dis 1988:11: 149-152.
53. Martin PJ, Hansen JA, Buckner CD, Sanders JE, Deeg HJ, Stewart P, Appelbaum FR, Clift R, Fefer A, Witherspoon RP, Kennedy MS, Sullivan KM, Flournoy N, Storb R, Thomas ED: Effects of in vitro depletion of T cells in HLA-identical allogeneic marrow grafts, Blood 1985:66:664-672.
54. Anasetti C, Martin PJ, Hansen JA, Appelbaum FR, Beatty PG, Doney K, Harkoncn S, Jackson A, Reichert T, Stewart P, Storb R, Sullivan KM, Thomas ED, Warner N, Witherspoon RP: A phase I- II study evaluating the murine anti-IL-2 receptor antibody 2A3 for treatment of acute graft-versus-host disease, Transplantation 1990;50:49-54.
55. Brown PS Jr., Parenteau GL, Dirbas FM, Garsia RJ, Goldman CK, Bukowski MA, Junghans RP, Queen C, Hakimi J, Benjamin WR, Clark RE, Waldmann TA: Anti-Tac-H, a humanized antibody to the interleukin 2 receptor, prolongs primate cardiac allograft survival, Proc Natl Acad Sei USA 1991:88: 2663-2667.
56. Ferrara JLM, Marion A, McIntyre JF, Murphy GF, Burakoff SJ: Amelioration of acute graft vs host disease due to minor histocompatibility antigens by in vivo administration of anti-interleukin 2 receptor antibody, J Immunol 1986:137:1874-1877.
57. Herve P, Wijdenes J, Bergerat JP, Bordigoni P, Milpied N, Cahn JY, Clement C, Beliard R, Morel-Fourrier B, Racadot E, Troussard X, Benz-Lemoine E, Gaud C, Legros M, Altai M, Kloft M, Peters A: Treatment of corticosteroid resistant acute graft-versus-host disease by in vivo administration of anti-intcrleukin-2 receptor monoclonal antibody (B-BIO), Blood 1990;75:1017-1023.
58. Anderson DM, Lyman SD, Baird A, Wignall JM, Eiscnman J, Rauch C, March CJ, Boswell IIS, Gimpel SD, Cosman D, Williams DE: Molecular cloning of mast cell growth factor, a hematopoictin that is active in both membrane bound and soluble forms, Cell 1990:63:235-243.
59. Deeg HJ, Storb R, Appclbaum FR, Kennedy MS, Graham TC, Thomas ED: Combined immunosuppression with cyclosporine and methotrexate in dogs given bone marrow grafts from DLA-haploidentical littermates, Transplantation 1984:37:62-65.
60. Martin PJ, Clift RA, Fisher LD, Buckner CD, Hansen JA, Appelbaum FR, Doney KC, Sullivan KM, Witherspoon RP, Storb R, Thomas ED: HLA-identi-cal marrow transplantation during accelerated phase chronic myelogenous leukemia: Analysis of survival and remission duration, Blood 1988;72:1978-1984.
61. Powles RL, Clink HM, Spence D, Morgenstern G, Watson JG, Selbv PJ, Woods M, Barrett A, Jameson B, Sloane J, Lawler SD, Kay HEM, Lawson D, McElwain TJ, Alexander P: Cyclosporin A to prevent graft-versus-host disease in man after allogeneic bone-marrow transplantation, Lancet 1980; 1:327-329.
62. Santos GW, Tutschka PJ, Brookmeycr R, Sarai R, Beschorner WE, Bias WB, Hayden GB, Burns WH, Farmer ER, Hess AD, Kaizer H, Mellits D, Sensen-brenner LL, Stuart R, Yeager AM: Cyclosporine plus methyiprednisolone versus cyclophosphamide plus methyiprednisolone as prophylaxis for graft-versus-host disease: A randomized double-blind study in patients undergoing allogeneic marrow transplantation, Clin Transplant 1987:1:21-28.
63. Storb R, Deeg HJ, Farewell V, Doney K, Appel-baum F, Beatty P, Bensinger W, Buckner CD, Dlift R, Hansen J, Hill R, Longton G, Lum L, Martin P, McGuffin R, Sanders J, Singer J, Stewart P, Sullivan K, Witherspoon R, Thomas HD: Marrow transplantation for severe aplastic anemia: Methotrexate alone compared with a combination of methotrexate and cyclosporine for prevention of acute graft-versus-host disease, Blood 1986:68:119-125.
64. Storb R, Deeg HJ, Pepe M, Appelbaum FR, Anasetti C, Beatty P, Bensinger W, Berenson R, Buckner CD, Dlift R, Doney K, Longton G, Hansen J, Hill R, Loughran T Jr., Martin P, Singer J, Sanders J, Stewart P, Sullivan K, Witherspoon R, Thomas ED: Methotrexate and cyclosporine versus cyclosporine alone for prophylaxis of graft-versus-host disease in patients given FI LA-identical marrow grafts for leukemia: Long term follow up of a controlled trial, Blood 1989:73:1729-1734.
65. Storb R, Deeg HJ, Pepe M, Doney K, Appelbaum F, Beatty P, Bensinger W, Buckner CD, Clift R, Hansen J, Ifill R, Longton G, Anasetti C, Martin P, Loughran IP, Sanders J, Singer J, Stewart P, Sullivan KM, Witherspoon R, Thomas ED: Graft-versus-host disease prevention by methotrexate combined with cyclosporine compared to methotrexate alone in patients given marrow grafts for severe aplastic anaemia: Long-term follow-up of a controlled trial, Br J Haematol 1989;72:567-572.
66. Storb R, Deeg FIJ, Whitehead J, Appelbaum F, Beatty P, Bensinger W, Buckner CD, Dlift R, Doney K, Farewell V, Hansen J, Flill R, Lum L, Martin P, McGuffin R, Sanders J, Stewart P, Sullivan K, Witherspoon R, Yee G, Thomas ED: Methotrexate and cyclosporine compared with cyclosporine alone for prophylaxis of acute graft versus host disease after marrow transplantation for leukemia, N Engl J Med 1986:314:729-735.
67. Maraninchi D, Gluckman L, Blaise D, Guyotat D, Rio B, Pico JL, Leblond V, Michaile! M, Dreyfus F, Ifrah N, Bordigoni A: Impact of T-cell depletion on outcome of allogeneic bone-marrow transplantation for standard-risk leukaemias, Lancet 1987;2: 175-178.
68. Martin PJ, Shulman HM, Schubach WH, Hansen JA, Fefer A, Miller G, Thomas ED: Fatal Epstein-Barr-virus-associated proliferation of donor B cells after treatment of acute graft-versus-host disease with a murine monoclonal anti-T-cell antibody, Ann Intern Med 1984:101:310-315.
69. Apperley JF, Jones L, Hale G, Waldmann H, Ilows J, Rombos Y, Tsatalas C, Marcus RE, Goolden AWG, Gordon-Smith EC, Catovsky D, Gallon DAG, Goldman JM: Bone marrow transplantation for patients with chronic myeloid leukaemia: T-cell depletion with Campath-1 reduces the incidence of graft-versus-host disease but may increase the risk of leukae-mic relapse, Bone Marrow Transplant 1986:1:53-66.
70. Goldman JM, Gale RP, Horowitz MM, Biggs JC, Champlin RE, Gluckman E, Hoffmann RG, Jacobsen SJ, Marmont AM, McGlave PB, Messner HA, Rimm AA, Rozman C, Speck B, Tura S, Weiner RS, Bortin MM: Bone marrow transplantation for chronic myelogenous leukemia in chronic phase: Increased risk of relapse associated with T-cell depletion, Ann Intern Med 1988:108.806-814.
71. Jacobson LG, Marks EK, Robson MJ, Gaston EG, Zirkle RE: Effect of spleen protection on mortality following x-irradiation, J Lab Clin Med 1949:34: 1538-1543.
72. Lorenz E, Uphoff D, Reid TR, Shelton E: Modification of irradiation injury in mice and guinea pigs by bone marrow injections, J Nall Cancer Inst 1951:12: 197-201.
73. Till JE, McCulloch EA: A direct measurement of the radiation sensitivity of normal mouse bone marrow cells, Radial Res 1961:14:213-222.
74. Becker AJ, McCulloch EA, Till JE: Cytological demonstration of the clonal nature of spleen colonies derived from transplanted mouse marrow cells, Nature 1963;197:452-454.
75. Siminovitch L, McCulloch EA, Till JE: The distribution of colony-forming cells among spleen colonies, J Cell Comp Physiol 1963;62:327-336.
76. Smith LG, Weissman IL, Heimfeld S: Clonal analysis of hematopoietic stem-cell differentiation in vivo, Proc Natl Acad Sei USA 1991:88:2788-2792.
77. Spangrude GJ: Hematopoietic stem-cell differentiation, C’urrOpin Immunol 1991:3:171-178.
78. w bone marrow cells repopulate the thymus, J Exp Med 1988;167:1671-1683.
79. Visser JW, Bauman JG, Mulder AH, Eliason JF, de Leeuw AM: Isolation of murine pluripotent hemopoietic stem cells, J Exp Med 1984:159:1576-1590.
80. Visser J WM, van Bekkum DW: Purification of pluripotent hemopoietic stem cells: Past and present, Exp Hcniatol 1990;18:248-256.
81. Muller-Sieburg CE, Whitlock CA, Weissman IL: Isolation of two early B lymphocyte progenitors from mouse marrow: A committed pre-pre-B cell and a clonogcnic Thy-1-low hematopoietic stem cell, Cell 1986:44:653-662.
82. Sutherland HJ, Eaves CJ, Eaves AC, Dragowska W, Lansdorp PM: Characterization and partial purification of human marrow cells capable of initiating long-term hematopoiesis in vitro, Blood 1989:74: 1563-1570.
83. w Lin-Sca-1 + cells are the only stem cells in C57BL/Ka-Thy-1,1 bone marrow, J Exp Med 1992:175:175-184.
84. Sour EF, Brandt JE, Briddel RA, Lccmhuis T, van Besien K, Hoffman R: Human CD34+DR- bone marrow cells contain progenitor cells capable of self-renewal, multilineage differentiation, and longterm in vitro hematopoiesis, Blood Cells 1991:17: 287-292.
85. Huang S, Terstappen LWMM: Formation of haematopoietic microenvironment and haematopoietic stem cells from single human bone marrow stem cells, Nature 1992:365:367-369.
86. Huang S, Terstappen LWMM: Formation of haematopoietic microenvironment and haematopoietic stem cells from single human bone marrow stem cells, Nature 1994:368:664 (correction).
87. Radka SF, Charron DJ, Brodsky FM: Review: Class II molecules of the major histocompatibility complex considered as differentiation markers, Hum Immunol 1986:16:390-400.
88. Huang S, Terstappen LWMM: Lymphoid and myeloid differentiation of single human CD34+, HLA-DR+, CD38- hematopoietic stem cells, Blood 1994: 83:1515-1526.
89. Srour EF, Brandi JE, Briddell RA, Grigsby S, Leemhuis T, Hoffman R: Long-term generation and expansion of human primitive hematopoietic progenitor cells in vitro, Blood 1993;81:661-669.
90. Broudy VC, Lin N, Zsebo KM, Birkett NC, Smith KA, Bernstein ID, Papavannopoulou T: Isolation and characterization of a monoclonal antibody that recognizes the human c-kit receptor, Blood 1992: 79:338-346.
91. Agrawal B, Manickasundari M, Fraga E, Singh B: T cells that recognize peptide sequences of self MHC class II molecules exist in syngeneic mice, J Immunol 1991:147:383-390.
92. Hogge DE, Sutherland HJ, Landsdorp PM, Phillips GL, Eaves CJ: The elusive peripheral blood hemopoietic stem cell, Semin Hcmatol 1993:30:82-89.
93. Kinniburgh D, Russell NH: Comparative study of CD34-positive cells and subpopulalions in human and umbilical cord blood and bone marrow, Bone Marrow Transplant 1993:12:489-494.
94. Huss R, Flong DS, McSweeney PA, Hoy CA, Deeg HJ: Differentiation of canine marrow cells with hematopoietic characteristics from an adherent stromal cell precursor, Proc Natl Acad Sci USA 1995:92:748-752.
95. Huss R, Floy CA, Deeg FIJ: Intercellular contact and growth factors involved in proliferation, differentiation and cell death in the bone marrow stroma, Blood 1995:85:2414-2421.
96. Heimfeld S, Hudak S, Weissman I, Rennick D: The in vitro response of phenotypically defined mouse stem cells and myeloervthroid progenitors to single or multiple growth factors, Proc Natl Acad Sci USA 1991;88:9902-9906.
97. Muller-Sieburg CE, Townsend K, Weissman IL, Rennick D: Proliferation and differentiation of highly enriched mouse hematopoietic stem cells and progenitor cells in response to defined growth factors, J Exp Med 1988:167:1825-1840.
98. Williams DE, Eiscnman J, Baird A, Rauch C, Van Ness K, March CJ, Park LS, Martin U, Moehizuki DY, Boswell HS, Burgess GS, Cosman D, Lyman SD: Identification of a ligand for the c-kit proto-oncogene, Cell 1990;63:167-174.
99. Zsebo KM, Williams DA, Geissler EN, Broudy VC, Martin FH, Atkins HL., Hsu R-Y, Birkett NC, Okino KH, Murdock DC, Jacobsen FW, Langley KF., Smith KA, Takeishi T, Cattanach BM, Galli SJ, Suggs SV: Stem cell factor is encoded at the SI locus of the mouse and is the ligand for the c-kil tyrosine kinase receptor, Cell 1990:63:213-224.
100. Chabot B, Stephenson DA, Chapman VM, Besmer F, Bernstein A: The proto-oncogene c-kit encoding a transmembrane tyrosine kinase receptor maps to the mouse Wlocus, Nature 1988:335:88-89.
101. Huss R, Hong D-S, Beckham C, Kimball L, Myer-son D, Storb R, Deeg HJ: Ultrastructural localization of stem cell factor in canine marrow-derived stromal cells, Exp J Lematol 1995:23:33-40.
102. Ikuta K, Weissman IL: Evidence that hematopoietic stem cells express mouse c-kil but do not depend on steel factor for their generation, Proc Natl Acad Sei USA 1992:89:1502-1506.
103. Okada S, Nakauchi H, Nagayoshi K, Nishikawa S, Nishikawa S, Miura Y, Suda T: Enrichment and characterization of murine hematopoietic stem cells that express c-kit molecule, Blood 1991:78: 1706-1712.
104. Capel B, Hawley RG, Mint B: Long- and shortlived murine hematopoietic stem cell clones individually identified with retroviral integration markers, Blood 1990;75:2267-2270.
105. Yarden Y, Kuang WJ, Yang-Feng T, Coussens L, Munemitsu S, Dull TJ, Chen E, Schlessinger J, Francke U, Ullrich A: Human proto-oncogene c-kit: A new cell surface receptor tyrosine kinase for an unidentified ligand, EMBO J 1987;6:3341-3351.
106. Andrews RG, Bartelmez SH, Knitter GH, Mverson D, Bernstein ID, Appelbaum FR, Zsebo KM: A c-kit ligand, recombinant human stem cell factor, mediates reversible expansion of multiple CD34+ colony-forming cell types in blood and marrow of baboons, Blood 1992;80:920-927.
107. Fleming WH, Alpern FJ, Uchida N, Ikuta K, Weissman IL: Steel factor influences the distribution and activity of murine hematopoietic stem cells in vivo, Proc Natl Acad Sei USA 1993;90:3760-3764.
108. Migliaccio G, Migliaccio AR, Druzin ML, Giardina PJ, Zsebo KM, Adamson JW: Effects of recombinant human stem cell factor (SCF) on the growth of human progenitor cells in vitro, J Cell Physiol 1991:148:503-509.
109. Novak U, Harris EA, Forrester W, Groudine M, Gelinas R: High-level b-globin expression after retroviral transfer of locus activation region-containing human b-globin gene derivatives into murine erythroleukemia cells, Proc Natl Acad Sei USA 1990:87:3386-3390.
110. Bernstein ID, Andrews RG, Zsebo KM: Recombinant human stem cell factor enhances the formation of colonies by CD34+ and CD34+lin-cells, and the generation of colony-forming cell progeny from CD34+lin-cells cultured with interleukin-3 (IL-3), granulocyte-macrophage colony-stimulating factor (GM-CSF), or granulocyte colony stimulating factor (G-CSF), Blood (in press).
111. McNiece IK, Langley KE, Zsebo KM: Recombinant human stem cell factor synergises with GM-CSF, G-CSF, IL-3 and epo to stimulate human progenitor cells of the myeloid and ervthroid lineages, Exp Hematol 1991:119:226-231.
112. Thomas ED, Storb R: Technique for human marrow grafting, Blood 1970:36:507-515.
113. Thomas ED, Storb R, Clift RA, Fefer A, Johnson FL, Neiman PE, Lerner KG, Glucksberg H, Buckner CD: Bone-marrow transplantation, N Engl J Med 1975:292:832-843,895-902.
114. Deeg HJ, Klingemann HG, Phillips GL: A Guide to Bone Marrow Transplantation, Berlin.Springer, 1988.
115. Kernan NA, Flomenberg N, Dupont B, O’Reilly RJ: Graft rejection in recipients of T-cell-depletcd HLA-nonidentical marrow transplants for leukemia, Transplantation 1987;43:842-847.
116. Sandell L., Johnson G, Przepiorka D, Torok-Storb B: Phenotype and function of T-cells associated with marrow graft failure and rejection: in Martelli MF, Grignani F, Reisner Y (eds): T-Cell Depletion in Allogeneic Bone-Marrow Transplantation, Serono Symposia Review No, 13, New York, Raven Press, 1988, pp 49-56.
117. Anderson KC, Barut BA, Ritz J, Freedman AS, Takvorian T, Ravinowe SN, Soiffcr R, Heflin L, Coral F, Dear K, Mauch P, Nadler LM: Monoclonal antibody-purged autologous bone marrow transplantation therapy for multiple myeloma, Blood 1991:77:712-720.
118. Foon KA, Tood RF III: Immunologic classification of leukemia and lymphoma, Blood 1986;68:1-31.
119. Areman EM, Deeg HJ, Sacher RA: Bone Marrow and Stem Cell Processing: A Manual of Current Techniques, Philadelphia, F.A, Davis & Co., 1992.
120. Baum CM, Weissman IL, Tsukamoto AS, Buckle A-M, Peault B: Isolation of a candidate human hematopoietic stem-cell population, Proc Natl Acad Sei USA 1992:89:2804.
121. Bender JG, Unverzagt K, Walker DE, Lee W, Smith S, Williams S, Van Epps DE: Phenotypic analysis and characterization of CD34+ cells from normal human bone marrow, cord blood, peripheral blood, and mobilized peripheral blood from patients undergoing autologous stem cell transplantation, Clin Immunol Immunopatho! 1994:70:10-18.
122. Brecher G, Cronkite EP: Post-radiation parabiosis and survival in rats, Proc Soc Exp Biol Med 1951:77:292-294.
123. Storb R, Epstein RB, Thomas ED: Marrow repopulating ability of peripheral blood cells compared to thoracic duct cells, Blood 1968:32:662-667.
124. Juttner CA, To LB, Haylock DN, Branford A, Kimber RJ: Circulating autologous stem cells collected in very early remission from acute non-Ivmphoblastic leukaemia produce prompt but incomplete haemopoietic reconstitution after high dose melphalan or supralclhal chemoradiotherapv, Br J Haematol 1985:61:739-745.
125. Lopez M, Mörtel O, Pouillart P, Zucker JM: Infusion of autologous peripheral blood nucleated cells hastens hematological recovery after high dose chemotherapy and autologous transplantation of bone marrow, Bone Marrow' Transplant 1990:5: 44-45.
126. Campos L, Bastion Y, Roubi N, Felman P, Espinouse D, Dumontel C, Debost M, Tremisi JP, Coiffier B: Peripheral blood stem cells harvested after chemotherapy and GM-CSF for treatment intensification in patients with advanced lympho-proliferative diseases, Leukemia 1993;7:1409-1415.
127. Siena S, Bregni M, Brando B, Ravagnani F, Bona-donna G, Gianni AM: Circulation of CD34+ hematopoietic stem cells in the peripheral blood of high-dose cyclophosphamide-treated patients: Enhancement by intravenous recombinant human granulocyte-macrophage colony-stimulating factor, Blood 1989:74:1905-1914.
128. Korbling M, Burke P, Braine FI, Elfenbein G, Santos GW, Kaizer H: Successful cngraftment of blood-derived normal hemopoietic stem cells in chronic myelogenous leukemia, Exp Hematol 1981;9:684-690.
129. Sharp JG, Armitage J, Crouse D, DeBoer J, Joshi S, Mann S, Weisenburger D, Kessinger A: Are occult tumor cells present in peripheral stem cell harvests of candidates for autologous transplantation? in Dicke KA, Spitzer G, Jagannath S, Evinger-Hodges MJ (eds): Autologous Bone Marrow Transplantation: Proceedings of the Fourth International Symposium, 1989, pp 693-696.
130. McNiece IK, Stewart FM, Deacon DM, Temelcs DS, Zsebo KM, Clark SC, Quesenbcrry PJ: Detection of a human CFC with a high proliferative potential, Blood 1989;74:609-612.
131. Russell NH, Hunter A, Rogers S, Hanley J, Anderson D: Peripheral blood stem cells as an alternative to marrow- for allogeneic transplantation, Lancet 1993:341:1482.
132. Rosenfeld CS, Cullis H, Tarosky T, Nemunaitis J: Peripheral blood stem cell collection using the small volume collection chamber in the Fenwal CS-30H Plus blood cell separator, Bone Marrow Transplant 1994;13:131-134.
133. Berenson RJ, Andrews RG, Bensinger WI, Kalamasz D, Knitter G, Buckner CD, Bernstein ID: Anligen CD34+ marrow cells engraft Icthaliy irradiated baboons, J Clin Invest 1988:81:951-955.
134. O’Reilly RJ, Pollack MS, Kapoor N, Kirkpatrick D, Dupont B: Fetal liver transplantation in man and animals: in Gale RP (ed): Recent Advances in Bone Marrow Transplantation, New York, Alan R, L.iss, 1983, pp 799-830.
135. Tourane JL: Transplantation of both fetal liver and thymus in severe combined immunodeficiencies: Interaction between donor’s and recipient's cells: in Lucarelli G, Fliedner TM, Gale RP (eds): Fetal Liver Transplantation, Amsterdam, Excerpta Medica, 1980, pp 276-283.
136. Harris DT, Schumacher MJ, Rvchlik S, Booth A, Acevedo A, Rubinstein P, Bard J, Boyse EA: Collection, separation and cryopreservation of umbilical cord blood for use in transplantation, Bone Marrow Transplant 1994:13:135-143.
137. Broxmeyer HE, Douglas GW, Hangoc G, Cooper S, Bard J, English D, Arnv M, Thomas L, Boyse EA: Human umbilical cord blood as a potential source of transplantable hematopoietic stem/progenitor cells, Proc Natl Acad Sei USA 1989:86: 3828-3832.
138. Gluckman E: Umbilical cord blood bank for transplantation of hematopoietic stem cells in man, Nouv Rev Fr Hematol 1993:35:293-294.
139. Gluckman E, Broxmeyer HE, Auerbach AD, Friedman HS, Douglas GW, Devergie A, Esperou II, Thierry D, Socie G, Lehn P, Cooper S, English D, Kurtzberg J, Bard J, Boyse EA: Hematopoietic reconstitution in a patient with Fanconi’s anemia by means of umbilical-cord blood from an HLA-identical sibling, N Engl J Med 1989:321:1174-1178.
140. Brugger W, Mocklin W, Heimfeld S, Berenson RJ, Mertelsmann R, Kanz L: Ex vivo expansion of enriched peripheral blood CD34+ progenitor cells by stem cell factor, interleukin-1 beta (IL-1 beta), 11.-6, IL-3, interfcron-gamma, and erythropoietin, Blood 1993:81:2579-2584.
141. Sato N, Sawada K, Koizumi K, Tarumi T, Ieko M, Yasukouchi T, Yamaguchi M, Takahashi TA, Sekiguchi S, Koike T: In vitro expansion of human peripheral blood CD34+ cells, Blood 1993:82: 3600-3609.
142. Smith LH, Phan IT: Long term preservation of bone marrow, Nature 1965:205:503-504.
143. Cavins JA, Kasakura S, Thomas ED, Ferrebee JW: Recovery of lethally irradiated dogs following infusion of autologous marrow stored at low temperature in dimethyl-sulphoxide, Blood 1962:20: 730-734.
144. Leibo SP, Farranl J, Mazur P, Hanna MG Jr., Smith LH: Effects of freezing on marrow stem cell suspensions: Interactions of cooling and warming rates:n the presence of PVP, sucrose, or glycerol, Cryobiology 1970:6:315-332.
145. Allien MA: Establishment of an AML-CFU colony assay to evaluate the sensitivity of leukemic progenitors to cryopreservation; in Incidence for Bone Marrow' Transplantation, Exp Hematol Today, New York, Springer, 1989, pp 69-71.
146. Douay L, Lopez M, Gorin NC, Nauman A, Giarratana MC, Laporte JP, Stachowiak J, Salmon C, Duhamel G: Failure of bone marrow cryopreservation in chronic granulocytic leukemia: Relation to excessive granulo-macrophagic progenitor pool, Int J Cell Cloning 1986;4:250-262.
147. Miller AD, Buttimore C: Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production, Mol Cell Biol 1986:6: 2895-2902.
148. Miller DG, Adam MA, Miller AD: Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection, Mol Cell Biol 1990;10:4239-4242.
149. Dunbar CE, Emmons RV: Gene transfer into hematopoietic progenitor and stem cells: Progress and problems, Stem Cells (Dayt) 1994;12:563-576.
150. Rolling F, Samulski RJ: AAV as a viral vector for human gene therapy, Generation of recombinant virus, Mol Biotechnol 1994:3:9-15.
151. Williams DA, Orkin SH, Mulligan RC: Retrovirus mediated gene transfer of human adenosine deaminase gene sequences into cells in culture and into murine hematopoietic cells in vivo, Proc Natl Acad Sci USA 1986;83:2566-2570.
152. Joyner A, Keller G, Phillips RA, Bernstein A: Retrovirus transfer of a bacterial gene into mouse haematopoietic progenitor cells, Nature 1983; 305:556-558.
153. Miller AD, Ong ES, Rosenfield MG, Verma IM, Evans RM: Infectious and selectable retrovirus containing an inducible rat growth hormone mini-gene, Science 1984:225:993-998.
154. Dick JE, Magli MC, Phillips RA, Berstein A, Caskey D: Expression of human adenosine deaminase gene in murine hematopoietic progenitor cells following retroviral transfer, Nature 1986: 322:385-387.
155. Hodgson GS, Bradley TR: Properties of hematopoietic stem cells surviving 5-fluorouracil treatment: Evidence for a pro CFU-S cell? Nature 1979:281:381.
156. Bowtell DDL, Cory S, Johnson GR, Gonda TJ: Comparison of expression in hematopoietic cells by retroviral vectors carrying two genes, J Virol 1988:62:2464-2473.
157. Eglilis MA, Kantoff PW, Jolly ID, Jones IB, Anderson WF, Lothrop CD Jr.: Gene transfer into hematopoietic progenitor cells from normal and cyclic hematopoietic dogs using retroviral vectors, Blood 1988:71:717-722.
158. Keller G, Paige D, Gilboa E, Wagner EF: Expression of a foreign gene in myeloid and lymphoid cells derived from multipolent hematopoietic precursors, Nature 1985:318:149-154.
159. Lcmischka IR, Raulet DH, Mulligan RC: Developmental potential and dynamic behavior of hematopoietic stem cells, Cell 1986:45:917-927.
160. Snodgrass R, Keller G: Clonal fluctuation within the hematopoietic system of mice reconstituted with retrovirus-infected stem cells, EMBO J 1987; 6:3955-3960.
161. Dick JE, Magli MC, Huszar D, Phillips RA, Bernstein A: Introduction of a selectable gene into primitive stem cells capable of long-term reconstitution of the hemopoietic system of W/Wv mice, Cell 1985:42:71-79.
162. Jordan CT, McKearn JP, Lcmischka IR: Cellular and developmental properties of fetal hematopoietic stem cells, Cell 1990;61:953-963.
163. Kwok WW, Schuening F, Stead RB, Miller AD: Retroviral transfer of genes into canine hemopoietic progenitor cells in culture: A model for human gene therapy, Proc Natl Acad Sci USA 1986; 83:4552-4555.
164. Stead RB, Kwok WW, Storb R, Miller AD: Canine model for gene therapy: Inefficient gene expression in dogs reconstituted with autologous marrow infected with retroviral vectors, Blood 1988;71: 742-747.
165. Eglilis MA, Kantoff PW, Jolly JD, Jones JB, Anderson WF, Lothrop CD: Gene transfer into hematopoietic progenitor cells from normal anti cyclic hematopoietic dogs using retroviral vectors, Blood 1988:71:717-722.
166. Kantoff PW, Flake AW, Eglitis MA, Scharf S, Bond S, Gilboa E, Erlich H, Harrison MR, Zanjani ED, Anderson WF: In utero gene transfer and expression: A sheep transplantation model, Blood 1989: 73:1066-1073.
167. Ekhterae D, Crumbleholme T, Karson E, Harrison MR, Anderson WF, Zanjani ED: Retroviral vector-mediated transfer of the bacterial neomycin resistance gene into fetal and adult sheep and human hematopoietic progenitors in vitro, Blood 1990:75:365-369.
168. Schuening FG, Storb R, Stead RB, Goehle S, Nash R, Miller AD: Improved retroviral transfer of genes into canine hematopoietic progenitor cells kept in long-term marrow culture, Blood 1989:74: 152-155.
169. Schuening FG, Kawahara K, Miller AD, To R, Goehle S, Stewart D, Mullally K, Graham TC, Appelbaum FR, Hackman R, Osborne WRA, Storb R: Retrovirus-mediated gene transduction into long-term repopulating marrow cells of dogs, Blood 1991:78:2568-2576.
170. Bodine AM, McDonagh KT, Brandt SJ, Ney PA, Agricola B, Byrne E, Nienhuis AW: Development of a high-titer retrovirus producer cell line capable of gene transfer into rhesus monkey hematopoietic stem cells, Proc Natl Acad Sci USA 1990:87: 3738-3742.
171. Carter RF, Abrams-Ogg ACG, Dick JE, Kruth SA, Valli VE, Kamel-Reid S, Dube ID: Autologous transplantation of canine long-term marrow culture cells genetically marked by retroviral vectors, Blood 1992:79:356-364.
172. Kiem HP, Darovskv B, von Kalle D, Goehle S, Stewart D, Graham T, Hackman R, Appelbaum FR, Deeg HJ, Miller AD, Storb R, Schuening FR: Retorvirus-mediated gene transduction into canine peripheral blood repopulaling cells, Blood 1994: 83:1467-1473.
173. Hock RA, Miller AD: Retrovirus-mediated transfer and expression of drug resistance genes in human haematopoietic progenitor cells, Nature 1986:320:275-277.
174. Hogge DE, Humphries RK: Gene transfer to primary normal and malignant human hemopoietic progenitors using recombinant retroviruses, Blood 1987:69:611-617.
175. Laneuville P, Chang W, Kamel-Reid S, Fauser A A, Dick JE: High-efficiency gene transfer and expression in normal human hematopoietic cells with retrovirus vectors, Blood 1988:71:811-814.
176. Bordignon C, Yu SF, Smith CA, Hantzopoulos P, Ungers GE, Keevcr CA, O'Reilly RJ, Gilboa E: Retroviral vector-mediated high-efficiency expression of adenosine deaminase (ADA) in hematopoietic long-term cultures of ADA-deficient marrow cells, Proc Natl Acad Sci USA 1989:86: 6748-6752.
177. Cournoyer D, Scarpa M, Mitani K, Moore KA, Markowitz D, Bank A, Belmont JW, Caskey CT: Gene transfer of adenosine deaminase into primitive human hematopoietic progenitor cells, Hum GeneTher 1991;2:203-213.
178. Nolta JA, Sender LS, Barranger JA, Kohn DB: Expression of human glucocerebrosidase in murine long-term bone marrow cultures after retroviral vector-mediated transfer, Blood 1990;75:787-797.
179. Crooks GM, Kohn DB: Growth factors increase amphotropic retrovirus binding to human CD34+ bone marrow progenitor cells, Blood 1993:82: 3290-3297.
180. Brenner MK, Rill DR, Holladay MS, Heslop HE, Moen RC, Busehlc M, Krance RA, Santana VM, Anderson WF, Ihle JN: Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients, Lancet 1993:342:1134-1137.
181. Dunbar CE, Bodine DM, Sorrentino B, Donahue R, Mconagh K, Cottler-Fox M, O'Shaughnessy J, Cowan K, Carter C, Doren S: Gene transfer into hematopoietic cells, Implications for cancer therapy, Ann N Y Acad Sci 1994:716:216-224.
182. Freeman SM, Whartenby KA, Abraham GN, Zwiebel JA: Clinical trials in gene therapy, Adv DrugDeliv Rev 1993;12:169-183.
183. Iiirschhorn R: Overview of biochemical abnormalities and molecular genetics of adenosine deaminase deficiency, PediatrRes 1993;3(1 suppl):S35-41.
184. Mclvor RS, Johnson MJ, Miller AD, Pitts S, Williams SR, Valerio D, Martin DW Jr, Verma IM: Human purine nucleoside phosphorylasc and adenosine deaminase: Gene transfer into cultured cells and murine hematopoietic stem cells by using recombinant amphotropic retroviruses, Mol Cell Biol 1987:7:838-846.
185. Valerio D, Duyvesteyn MG, vander Eb AJ: Introduction of sequences encoding functional human adenosine deaminase into mouse cells using a retroviral shuttle system, Gene 1985;34:163-168.
186. Kantoff PW, Kohn DB, Mitsuya H, Armentano D, Sieberg M, Zwiebel JA, Eglitis MA, McLachlin JR, Wiginton DA, Hutton JJ, et al: Correction of adenosine deaminase deficiency in cultured human T and B cells by retrovirus-mediated gene transfer, Proc Natl Acad Sci USA 1986;83:6563-6567.
187. Kantoff PW, Gillio AP, McLachlin JR, Bordignon C, Eglitis MA, Kernan NA, Moen RC, Kohn DB, Yu SF, Karson E, Karlsson S, Zwiebel JA, Gilboa E, Bkiese RM, Nienhuis A, O'Reilly RJ, Anderson WF: Expression of human adenosine deaminase in nonhuman primates after retrovirus-mediated gene transfer, J Exp Med 1987:166:219-234.
188. Lim B, Apperley JF, Orkin SH, Williams DA: Long-term expression of human adenosine deaminase in mice transplanted with retrovirus-infected hematopoietic stem cells, Proc Natl Acad Sci USA 1989:86:8892-8896.
189. Kaleko M, Garcia JV, Osborne WRA, Miller AD: Expression of human adenosine deaminase in mice after transplantation of genetically-modified bone marrow, Blood 1990;75:1733-1741.
190. Osborne WRA, Hock RA, Kaleko M, Miller AD: Long-term expression of human adenosine deaminase in mice after transplantation of bone marrow infected with amphotropic retroviral vectors, Hum GeneTher 1990;1:31-41.
191. Einerhand MP, Bakx TA, Kukler A, Valerio D: Factors affecting the transduction of pluripotent hematopoietic stem cells: Long-term expression of a human adenosine deaminase gene in mice, Blood 1993:81:254-263.
192. Bodine DM, Moritz T, Donahue RE, Luskey BD, Kessler SW, Marlin Dl, Orkin SH, Nienhuis AW, Williams DA: 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 1993:82:1975-1980.
193. van Bcusechem VW, Kukler A, Heidt PJ, Valerio D: Long-term expression of human adenosine deaminase in rhesus monkeys transplanted with retrovirus-infected bone-marrow cells, Proc Natl Acad Sci USA 1992:89:7640-7644.
194. Kohn DB, Weinberg KI, Park man R, Lenarsky D, Crooks GM, Shaw K, Hanley ME, Lawrence K, Annett G, Brooks JS, Wara D, Elder M, Bowen T, Hershfield MS, Berenson RI, Moen RC, Mullen CA, Blaese RM: Gene therapy for neonates with ADA-deficient SC1D by relroviral-mediated transfer of the human ADA cDNA into umbilical cord CD34+ cells (abstr), Blood 1993:82(Supplement 1):315a.
195. Hoogerbrugge PM, van Beusechem V, Valerio D, Moseley A, Harvey M, Fischer A, Debree M, Ciaspar B, Morgan G, Levinsky R: Gene therapy in 3 children with adenosine deaminase deficiency (abstr), Blood 1993;82(Supplement 1):315a.
196. Steinberg MH: Prospects of gene therapy for hemoglobinopathies, Am J Med Sci 1991:302: 298-303.
197. Grosveld F, van Assendelft GB, Greaves DR, Kollias D: Position-independent, high-level expression of the human b-globin gene in transgenic mice, Cell 1987:51:975-985.
198. Orkin SH: Globin gene regulation and switching: Circa 1990, Cell 1990;63:665-672.
199. Einerhand MP, Antoniou M, Zolotukhin S, Muzyczka N, Berns KI, Grosveld F, Valerio D: Regulated high-level human beta-globin gene expression in ervthroid cells following recombinant adeno-associated virus-mediated gene transfer, GeneTher 1995:2:336-343.
200. Bender MA, Gelinas RE, Miller AD: A majority of mice show long-term expression of a human b-globin gene after retrovirus transfer into hematopoietic stem cells, Mol Cell Biol 1989:9:1426-1434.
201. Bodine DM, Karlsson S, Nienhuis AW: Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells, Proc Natl Acad Sci USA 1989;86:8897-8901.
202. Dzierzak EA, Papayannopoulou T, Mulligan RC: Lineage-specific expression of a human a-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells, Nature 1988;331:35-41.
203. Powars DR, Chan L, Schroeder WA: The influence of fetal hemoglobin on the clinical expression of sickle cell anemia, Ann N Y Acad Sci 1989:565: 262-278.
204. Charache S, Dover GJ, Moyer MA, Moore JW: Hydroxyurea-induced augmentation of fetal hemoglobin production in patients with sickle cell anemia, Blood 1987:69:109-116.
205. Sorge J, Kuhl W, West C, Beutler E: Complete correction of the enzymatic defect of type 1 Gaucher disease fibroblasts by retroviral-mediated gene transfer, Proc Natl Acad Sci USA 1987:84:906-909.
206. Weinthal J, Nolta JA, Yu XJ, Lilley J, Uribe L, Kohn DB: Expression of human glucocerebrosidase following retroviral vector-mediated transduction of murine hematopoietic stem cells, Bone Marrow Transplant 1991;8:403-412.
207. Ohashi T, Boggs S, Robbins P, Bahnson A, Patrene K, Wei FS, Wei JF, Li J, Lucht L, Fei Y, Clark S, Kimak M, He H, Mowery-Rush ton P, Barrangcr JA: Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector, Proc Natl Acad Sci USA 1992:89: 11332-11336.
208. Fink JK, Correll PH, Perry LK, Brady RO, Karlsson S: Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease, Proc Nat Acad Sci USA 1990; 87:2334-2338.
209. Nolta JA, Yu XJ, Bahner I, Kohn DB: Retroviral-mediated transfer of the human glucocerebrosidase gene into cultured Gaucher bone marrow, J Clin Invest 1992:90:342-348.
210. Xu L, Stahl SK, Dave HPG, Schiffman R, Correll PH, Kessler S, Karlsson S: Correction of the enzyme deficiency in hematopoietic cells of Gaucher patients using a clinically acceptable retroviral supernatant transduction protocol, Exp Hematol 1994:22:223-230.
211. von Kalle C, Kieni H-P, Barquinero J, Darovsky B, Duboikin M, Hcimfcld S, Barringer J: Transfer of the human glucocerebrosidase gene into canine and human repopulating cells (abstr), J Cell Bio-chem 1994:suppl 18A:230.
212. Leavitt MC, Yu M, Yamada O, Kraus G, Looney D, Poeschla E, Wong-Staal F: Transfer of an anti-HIV-1 ribozyme gene into primary human lymphocytes, Hum Gen Thcr 1994:5:1115-1120.
213. Lisziewicz J, Sun D, Lisziewicz A, Gallo RC: Anti-tat gene therapy: A candidate for late-stage AIDS patients, Gene Ther 1995;2:218-222.
214. Woffendin C, Yang ZY, Udaykumar, Xu L, Yang NS, Sheehy MJ, Nabel GJ: Nonviral and viral delivery of a human immunodeficiency virus protective gene into primary human T cells, Proc Natl Acad Sci USA 1994;91:11581-11585.
215. Lever AM: Gene therapy for HIV infection, Gr Med Bull 1995:51:149-166.
216. Zicgner UH, Peters G Joly DJ, Mento SJ, Galpin J, Prussak CE, Barber JR, Hartnett DE, Bohart C, Klump W: Cytotoxic T-lymphocytes induction in asymptomatic HIV-1-infected patients immunized with Retrovector-transduced autologous fibroblasts expressing HIV-1 111B Env/Rev proteins, AIDS 1995:9:43-50.
217. Sorrentino BP, Brandt SJ, Bodine D, Gottesman M, Pastan I, Cline A, Nienhuis AW: Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1, Science 1992; 257:99-103.
218. Gottesman MM: The multidrug resistance gene as a selectable marker in gene therapy, Recent Advances in Hematopoietic Stem Cell Transplantation, Clinical Progress, New Technologies and Gene llierapy, San Diego, CA, March 10-12,1994.
219. Brenner MK, Rill DR, Moen RC, Krance RA, Mirro J Jr., Anderson WF, Ihle JN: Gene-marking to trf.ee origin of relapse after autologous bone-marrow transplantation, Lancet 1993:341:85-86.
220. Deisseroth A, Zu Z, Claxton D, Ilanania E, Berenson R, Heimfeld S, Brenner M, Moen R, Anderson WF, Talpaz M, Hamer J, Calvert L, Ellerson D, Goldberg L, Janicek K, Thomas M, Kantarjian H, Champlin R, Reading C: Retroviral marking studies show that infused exogenous marrow cells give rise to hematopoietic recovery after autologous transplant, and that relapse after auto-logous transplant in CM I, may arise from Ph+ cells present in marrow at the time of transplant (abstr), Blood 1993:82(suppl 1):454a.
221. Elshami AA, Kucharczuk JC, Sterman DH, Smythe WR, Hwang HC, Amin KM, Litzky LA, Albeda SM, Kaiser LR: The role of immunosuppression in the efficacy of cancer gene therapy using adenovirus transfer of the herpes simplex thvm dine kinase gene, Ann Surg 1995:222: 298-307.
222. Blaese RM, Ishii-Morita H, Mullen C, Ramsey J, Ram Z, Oldfield E, Culver K: In situ delivery of suicide genes for cancer treatment, Eur J Cancer 1994: 30A: 1187-1190.
223. Aoki K, Yoshida T, Sugimura T, Terada M: Liposome-mediated in vivo gene transfer of antisense K-ras construct inhibits pancreatic tumor dissemination in the murine peritoneal cavity, Cancer Res 1995:55:3810-3816.
224. Cornetta K, Moore A, Johannessohn M, Sledge GW: Clonal dominance detected in métastasés but not primary tumors of retrovirally marked human breast carcinomas injected into nude mice, Clin Exp Metastasis 1994:12:3-12.