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References

  • 1
    Williams DA, Nienhuis AW, Hawley RG et al. Gene therapy 2000. In: SchechterD, ed. American Society of Hematology Education Program. Philadelphia: W.B. Saunders, 2000:376393.
  • 2
    Karlsson S. Treatment of genetic defects in hematopoietic cell function by gene transfer. Blood 1991; 78:24812492.
  • 3
    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; 10:42394242.
  • 4
    Richter J. Gene transfer to hematopoietic cells: The clinical experience. Eur J Haematol 1997; 59:6775.
  • 5
    Gothot A, Van Der Loo JC, Clapp DW et al. Cell cycle-related changes in repopulating capacity of human mobilized peripheral blood CD341 cells in non-obese diabetic/severe combined immune-deficient mice. Blood 1998; 92:26412649.
  • 6
    Guenechea G, Segovia JC, Albella B et al. Delayed engraftment of nonobese diabetic/severe combined immunodeficient mice transplanted with ex vivo-expanded human CD341 cord blood cells. Blood 1999; 93:10971105.
  • 7
    Conneally E, Cashman J, Petzer A et al. Expansion in vitro of transplantable human cord blood stem cells demonstrated using a quantitative assay of their lympho-myeloid repopulating activity in non obese diabetic-scid/scid mice. Proc Natl Acad Sci U S A 1997; 94:98369841.
  • 8
    Piacibello W, Sanavio F, Garetto L et al. Extensive amplification and self-renewal of human primitive hematopoietic stem cells from cord blood. Blood 1997; 89:26442653.
  • 9
    Kollet O, Aviram R, Chevath J et al. The soluble interleukin-6 (IL-6) receptor/IL-6 fusion protein enhances in vitro maintenance and proliferation of human CD34(+)CD38(+/Low) cells capable of repopulating severe combined immunodeficiency mice. Blood 1999; 94:923931.
  • 10
    Piacibello W, Sanavio F, Severino A et al. Engraftment in non obese diabetic severe combined immunodeficient mice of human CD34(+) cord blood cells after ex vivo expansion: Evidence for the amplification and self-renewal of repopulating stem cells. Blood 1999; 93:37363749.
  • 11
    Ueda T, Tsuji K, Yoshino H et al. Expansion of human NOD/SCID-repopulating cells by stem cell factor, Flk2/Flt3 ligand, thrombopoietin, IL-6, and soluble IL-6 receptor. J Clin Invest 2000; 105:10131021.
  • 12
    Gilmore GL, Depasquale DK, Lister J et al. Ex vivo expansion of human umbilical cord blood CD34(+) hematopoietic stem cells. Exp Hematol 2000; 28:297305.
  • 13
    Lazzari L, Lucchi S, Rebulla P et al. Long-term expansion and maintenance of cord blood haematopoietic stem cells using thrombopoietin, Flt3-ligand, interleukin (IL)-6 and IL-11 in a serum-free and stroma-free culture system. Br J Haematol 2001; 112:397404.
  • 14
    Blundell MP, Demaison C, Brouns G et al. Quality of repopulation in nonobese diabetic severe combined immunodeficient mice engrafted with expanded cord blood CD34+ cells. Blood 1999; 94:32693270.
  • 15
    Larochelle A, Vormoor J, Lapidot T et al. Engraftment of immune-deficient mice with primitive hematopoietic cells from beta-thalassemia and sickle cell anemia patients: Implications for evaluating human gene therapy protocols. Hum Mol Genet 1995; 4:163172.
  • 16
    Lapidot T, Pflumio F, Doedens M et al. Cytokine stimulation of multilineage hematopoiesis from immature human cells engrafted in SCID mice. Science 1992; 255:24062411.
  • 17
    Cashman JD, Lapidot T, Wang JCY et al. Kinetic evidence of the regeneration of multilineage hematopoiesis from primitive cells in normal human bone marrow transplanted into immunodeficient mice. Blood 1997; 89:43074316.
  • 18
    Pflumio F, Izac B, Katz A et al. Phenotype and function of human hematopoietic cells engrafting immune-deficient CB 7-severe combined immunodeficiency mice and non obese diabetic-severe combined immunodeficiency mice after transplantation of human cord blood mononuclear cells. Blood 1996; 88:37313740.
  • 19
    Chevalier S, Praloran V, Smith C et al. Expression and functionality of the trkA protooncogene product/NGF receptor in undifferentiated hematopoietic cells. Blood 1994; 6:14791485.
  • 20
    Torcia M, Bracci-Laudiero L, Lucibello M et al. Nerve growth factor is an autocrine survival factor for memory B lymphocytes. Cell 1996; 85:354356.
  • 21
    Bonini C, Ferrari G, Verzeletti S et al. HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science 1997; 276:17191724.
  • 22
    Ruggieri L, Aiuti A, Salomoni M et al. Cell-surface marking of CD34+ restricted phenotypes of human hematopoietc progenitor cells by retrovirus-mediated gene transfer. Hum Gene Ther 1997; 8:16111623.
  • 23
    Giaretta I, Madeo D, Bonaguro R et al. A comparative evaluation of gene transfer into blood cells using the same retroviral backbone for independent expression of the EGFP and deltaLNFGR marker genes. Haematologica 2000; 85:680689.
  • 24
    Movassagh M, Desmyter C, Baillou C et al. High level gene transfer to cord blood progenitors using gibbon ape leukemia virus pseudotype retroviral vectors and an improved clinically applicable protocol. Hum Gene Ther 1998; 9:225234.
  • 25
    Gluckman E. Hematopoietic stem cell transplant using umbilical cord-blood. N Engl J Med 2001; 24:18601861.
  • 26
    Kohn D, Weinberg K, Nolta J et al. Engraftment of gene-modified umbilical cord blood cells in neonateds with adenoise deaminase deficiency. Nat Med 1995; 1:10171023.
  • 27
    Cavazzana-Calvo M, Hacein-Bey S, De Saint Basile G et al. Gene therapy of human severe combined immunodeficiency (Scid)-X1 disease. Science 2000; 28:627629.
  • 28
    Kiem HP, Heyward S, Winkler A et al. Gene transfer into marrow repopulating cells: Comparison between amphotropic and gibbon ape leukemia virus pseudotyped retroviral vectors in a competititve repopulation assay in baboons. Blood 1997; 90:46384645.
  • 29
    Miller AD, Garcia JV, von Suhr N et al. Construction and properties of retrovirus packaging cells based on gibbon ape leukemia virus. J Virol 1991; 65:22202224.
  • 30
    Kavanaugh MP, Miller DG, Zhang W et al. Cell-surface receptors for gibbon ape leukemia virus and amphotropic murine retrovirus are inducible sodium-dependent phosphate symporters. Proc Natl Acad Sci U S A 1994; 91:70717075.
  • 31
    McCowage GB, Phillips KL, Gentry TL et al. Multiparameter-fluorescence activated cell sorting analysis of retroviral vector gene transfer into primitive umbilical cord blood cells. Exp Hematol 1998; 26:288298.
  • 32
    Piacibello W, Sanavio F, Garetto L et al. Differential growth factor requirement of primitive cord blood hematopoietic stem cell for self-renewal and amplification vs proliferation and differentiation. Leukemia 1998; 12:718727.
  • 33
    Piacibello W, Garetto L, Sanavio F et al. The effects of FLT3 ligand on in vitro human megakaryocytopoiesis. Exp Hematol 1996; 24:340346.
  • 34
    Becker M, Nitsche A, Neumann C et al. Sensitive PCR method for the detection and real-time quantification of human cells in xenotransplantation systems. Br J Cancer 2002; 87:13281335.
  • 35
    Schilz AJ, Brouns G, Knoss H et al. High efficiency gene transfer to human hematopoietic SCID-repopulating cells under serum-free conditions. Blood 1998; 92:31633171.
  • 36
    Hao QL, Thiemann FT, Petersen D et al. Extended long-term culture reveals a highly quiescent and primitive human hematopoietic progenitor populatio. Blood 1996;88:33063313.
  • 37
    Glimm H, Kiem HP, Darovsky B et al. Efficient gene transfer in primitive CD34+/CD38lo human bone marrow cells reselected after long-term exposure to GALV-pseudotyped retroviral vector. Hum Gene Ther 1997; 8:20792086.
  • 38
    Vormoor J, Lapidot T, Pflumio F et al. Immature human cord blood progenitors engraft and proliferate to high levels in severe combined immunodeficient mice. Blood 1994; 83:24892497.
  • 39
    Bhatia M, Wang JCY, Kapp U et al. Purification of primitive human hematopoietic cells capable of repopulating immune-deficient mice. Proc Natl Acad Sci U S A 1997; 94:53205325.
  • 40
    Piacibello W, Bruno S, Sanavio F et al. Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient. Blood 2002; 100:43914400.
  • 41
    Fisher A, Cavazzana-Calvo M. Integration of retroviruses: A fine balance between efficiency and danger. PLoS Med 2005; 2:e10.
  • 42
    Gammaitoni L, Weisel KC, Gunetti M et al. Elevated telomerase activity and minimal telomere loss in cord blood long-term cultures with extensive stem cell replication. Blood 2004; 103:44404448.
  • 43
    Hacein-Bey-Abina S, Le Deist F, Carlier F et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002; 18:3461193.
  • 44
    Hacein-Bey-Abina S, von Kalle C, Schmidt M et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003; 348:255256.
  • 45
    Hacein-Bey-Abina S, Von Kalle C, Schmidt M et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302:415419.
  • 46
    Schmidt M, Hacein-Bey-Abina S, Wissler M et al. Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial. Blood 2005; 105:26992706.
  • 47
    Kiem HP, Sellers S, Thomasson B et al. Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: No progression to clonal hematopoiesis or leukemia. Mol Ther 2004; 9:389395.
  • 48
    Kustikova O, Fehse B, Modlich U et al. Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science 2005; 308:11711174.
  • 49
    De Palma M, Montini E, de Sio FR et al. Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. Blood 2005; 105:23072315.
  • 50
    Laufs S, Nagy KZ, Giordano FA et al. Insertion of retroviral vectors in NOD/SCID repopulating human peripheral blood progenitor cells occurs preferentially in the vicinity of transcription start regions and in introns. Mol Ther 2004; 10:874881.
  • 51
    Bonini C, Grez M, Traversari C et al. Safety of retroviral gene marking with a truncated NGF receptor. Nat Med 2003; 9:367369.
  • 52
    Mazurier F, Gan OI, McKenzie JL et al. Lentivector-mediated clonal tracking reveals intrinsic heterogeneity in the human hematopoietic stem cell compartment and culture-induced stem cell impairment. Blood 2004; 103:545552.