Seminars in Hematology
Volume 41, Issue 4 , Pages 257-271 , October 2004

Genetic manipulation of hematopoietic stem cells

  • André Larochelle

      Affiliations

    • Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    • ,
  • Cynthia E. Dunbar

      Affiliations

    • Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    • Corresponding Author InformationAddress correspondence to Cynthia E. Dunbar, MD, National Heart, Lung, and Blood Institute, National Institutes of Health, Building 10, Room 7C-103, 9000 Rockville Pike, Bethesda, MD 20892

References 

  1. Pages JC , Bru T . Toolbox for retrovectorologists . J Gene Med . 2004;6(suppl 1):S67–S82
  2. Donahue RE , Kessler SW , Bodine D , McDonagh K , Dunbar C , Goodman S , et al.   Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer . J Exp Med . 1992;176:1125–1135
  3. Chen J , Reeves L , Sanburn N , Croop J , Williams DA , Cornetta K . Packaging cell line DNA contamination of vector supernatants (Implication for laboratory and clinical research) . Virology . 2001;282:186–197
  4. Cavazzana-Calvo M , Hacein-Bey S , de Saint Basile G , Gross F , Yvon E , Nusbaum P , et al.   Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease . Science . 2000;288:669–672
  5. Hacein-Bey-Abina S , von Kalle C , Schmidt M , McCormack MP , Wulffraat N , Leboulch P , et al.   LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 . Science . 2003;302:415–419
  6. Hacein-Bey-Abina S , von Kalle C , Schmidt M , Le Deist F , Wulffraat N , McIntyre E , et al.   A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency . N Engl J Med . 2003;348:255–256
  7. Roe T-Y , Reynolds TC , Yu G , Brown PO . Integration of murine leukemia virus DNA depends on mitosis . EMBO J . 1993;12:2099–2108
  8. Nolta JA , Smogorzewska EM , Kohn DB . Analysis of optimal conditions for retroviral-mediated transduction of primitive human hematopoietic cells . Blood . 1995;86:101–105
  9. Glimm H , Oh IH , Eaves CJ . Human hematopoietic stem cells stimulated to proliferate in vitro lose engraftment potential during their S/G(2)/M transit and do not reenter G(0) . Blood . 2000;96:4185–4193
  10. Zufferey R , Dull T , Mandel RJ , Bukovsky A , Quiroz D , Naldini L , et al.   Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery . J Virol . 1998;72:9873–9880
  11. Ramezani A , Hawley TS , Hawley RG . Lentiviral vectors for enhanced gene expression in human hematopoietic cells . Mol Ther . 2000;2:458–469
  12. Salmon P , Kindler V , Ducrey O , Chapuis B , Zubler RH , Trono D . High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors . Blood . 2000;96:3392–3398
  13. Logan AC , Lutzko C , Kohn DB . Advances in lentiviral vector design for gene-modification of hematopoietic stem cells . Curr Opin Biotechnol . 2002;13:429–436
  14. May C , Rivella S , Callegari J , Heller G , Gaensler KML , Luzzatto L , et al.   Therapeuitc haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin . Nature . 2000;406:82–86
  15. Kowolik CM , Hu J , Yee JK . Locus control region of the human CD2 gene in a lentivirus vector confers position-independent transgene expression . J Virol . 2001;75:4641–4648
  16. Lutzko C , Peterson D , Senadheera D , Kohn DB . Regulated transgene expression in human and murine B-lymphoid cells from lentiviral vectors carrying the immunoglobulin heavy chain enhancer and MARs . Mol Ther . 2004;5(suppl):s425; (abstr)
  17. Cui Y , Golob J , Kelleher E , Ye Z , Pardoll D , Cheng L . Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells . Blood . 2002;99:399–408
  18. Josephson NC , Vassilopoulos G , Trobridge GD , Priestley GV , Wood BL , Papayannopoulou T , et al.   Transduction of human NOD/SCID-repopulating cells with both lymphoid and myeloid potential by foamy virus vectors . Proc Natl Acad Sci USA . 2002;99:8295–8300
  19. Vassilopoulos G , Josephson NC , Trobridge G . Development of foamy virus vectors . Methods Mol Med . 2003;76:545–564
  20. Bett AJ , Prevec L , Graham FL . Packaging capacity and stability of human adenovirus type 5 vectors . J Virol . 1993;67:5911–5921
  21. Srivastava A . Obstacles to human hematopoietic stem cell transduction by recombinant adeno-associated virus 2 vectors . J Cell Biochem . 2002;38(suppl):39–45
  22. Alexander IE , Russell DW , Miller AD . Transfer of contaminants in adeno-associated virus vector stocks can mimic transduction and lead to artifactual results . Hum Gene Ther . 1997;8:1911–1920
  23. Hargrove PW , Vanin EF , Kurtzman GJ , Nienhuis AW . High-level globin gene expression mediated by a recombinant adeno-associated virus genome that contains the 3′ gamma globin gene regulatory element and integrates as tandem copies in erythroid cells . Blood . 1997;89:2167–2175
  24. Nathwani AC , Hanawa H , Vandergriff J , Kelly P , Vanin EF , Nienhuis AW . Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38− subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV . Gene Ther . 2000;7:183–195
  25. Chatterjee S , Li W , Wong CA , Fisher-Adams G , Lu D , Guha M , et al.   Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adeno-associated virus vectors . Blood . 1999;93:1882–1894
  26. Yang Q , Mamounas M , Yu G , Kennedy S , Leaker B , Merson J , et al.   Development of novel cell surface CD34-targeted recombinant adenoassociated virus vectors for gene therapy . Hum Gene Ther . 1998;9:1929–1937
  27. Handa A , Muramatsu S , Qiu J , Mizukami H , Brown KE . Adeno-associated virus (AAV)-3-based vectors transduce haematopoietic cells not susceptible to transduction with AAV-2-based vectors . J Gen Virol . 2000;81:2077–2084
  28. Schmidt-Wolf GD , Schmidt-Wolf IG . Non-viral and hybrid vectors in human gene therapy (an update) . Trends Mol Med . 2003;9:67–72
  29. Mikkelsen JG , Yant SR , Meuse L , Huang Z , Xu H , Kay MA . Helper-independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo . Mol Ther . 2003;8:654–665
  30. Ivics Z , Hackett PB , Plasterk RH , Izsvak Z . Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells . Cell . 1997;91:501–510
  31. Dick JE , Magli MC , Huszar D , Phillips RA , Berstein 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
  32. Keller G , Paige C , Gilboa E , Wagner EF . Expression of a foriegn gene in myeloid and lymphoid cells derived from multipotent haematopoietic precursors . Nature . 1985;318:149–154
  33. Lemischka IR , Raulet DH , Mulligan RC . Developmental potential and dynamic behavior of hematopoietic stem cells . Cell . 1986;45:917–927
  34. Brenner MK , Rill DR , Holladay MS , Heslop HE , Moen RC , Buschle M , et al.   Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients . Lancet . 1993;342:1134–1137
  35. Deisseroth AB , Zu Z , Claxton D , Hanania EG , Fu S , Ellerson D , et al.   Genetic marking shows that Ph+ cell present in autologous transplants of chronic myelogenous leukemia (CML) contribute to relapse after autologous bone marrow transplantation in CML . Blood . 1994;83:3068–3076
  36. Dunbar CE , Cottler-Fox M , O’Shaughnessy J , Doren S , Carter CS , Berenson R , et al.   Retrovirally-marked CD34-enriched peripheral blood and bone marrow and cells contribute to long-term engraftment after autologous transplantation . Blood . 1995;85:3048–3057
  37. Rill DR , Santana VM , Roberts WM , Nilson T , Bowman LC , Krance RA , et al.   Direct demonstration that autologous bone marrow transplantation for solid tumors can return a multiplicity of tumorigenic cells . Blood . 1994;84:380–383
  38. Richter J , Karlsson S . Clinical gene therapy in hematology (Past and future) . Int J Hematol . 2001;73:162–169
  39. Zanjani ED , Pallavicini MG , Ascensao JL , Flake AW , Langlois RG , Reitsma M , et al.   Engraftment and long-term expression of human fetal hemopoietic stem cells in sheep following transplantation in utero . J Clin Invest . 1992;89:1178–1188
  40. Porada CD , Tran ND , Almeida-Porada G , Glimp HA , Pixley JS , Zhao Y , et al.   Transduction of long-term-engrafting human hematopoietic stem cells by retroviral vectors . Hum Gene Ther . 2002;13:867–879
  41. Kamel-Reid S , Dick JE . Engraftment of immune-deficient mice with human hematopoietic stem cells . Science . 1988;242:1706–1709
  42. Shultz LD , Schweitzer PA , Christianson SW , Gott B , Schweitzer IB , Tennent B , et al.   Multiple defects in innate and adaptive immunologic function in NOD/LtSz-scid mice . J Immunol . 1995;154:180–191
  43. Kollet O , Peled A , Byk T , Ben-Hur H , Greiner D , Schultz L , et al.   β2 Microglobulin-deficient (B2mnull) NOD/SCID mice are excellent recipients for studying human stem cell function . Blood . 2000;95:3102–3105
  44. Larochelle A , Vormoor J , Hanenberg H , Wang JCY , Bhatia M , Lapidot T , et al.   Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID mouse bone marrow (implications for gene therapy) . Nat Med . 1996;2:1329–1337
  45. Guenechea G , Gan OI , Dorrell C , Dick JE . Distinct classes of human stem cells that differ in proliferative and self-renewal potential . Nat Immunol . 2001;2:75–82
  46. Horn PA , Thomasson BM , Wood BL , Andrews RG , Morris JC , Kiem HP . Distinct hematopoietic stem/progenitor cell populations are responsible for repopulating NOD/SCID mice versus nonhuman primates . Blood . 2003;102:4329–4335
  47. Donahue RE , Dunbar CE . An update on the use of non-human primate models for preclinical testing of gene therapy approaches targeting hematopoietic cells . Hum Gene Ther . 2001;12:607–617
  48. Aiuti A , Slavin S , Aker M , Ficara F , Deola S , Mortellaro A , et al.   Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning . Science . 2002;296:2410–2413
  49. Hacein-Bey-Abina S , Le Deist F , Carlier F , Bouneaud C , Hue C , De Villartay JP , et al.   Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy . N Engl J Med . 2002;346:1185–1193
  50. Dunbar CE , Seidel NE , Doren S , Sellers S , Cline AP , Metzger ME , et al.   Improved retroviral gene transfer into murine and rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colony-stimulating factor . Proc Natl Acad Sci USA . 1996;93:11871–11876
  51. Habibian HK , Peters SO , Hsieh CC , Wuu J , Vergilis K , Grimaldi CI , et al.   The fluctuating phenotype of the lymphohematopoietic stem cell with cell cycle transit . J Exp Med . 1998;188:393–398
  52. Peters SO , Kittler ELW , Ramshaw HS , Quesenberry PJ . Murine marrow cells expanded in culture with IL-3, IL-6, IL-11, and SCF acquire an engraftment defect in normal hosts . Exp Hematol . 1995;23:461–466
  53. Tisdale JF , Hanazono Y , Sellers SE , Agricola BA , Metzger ME , Donahue RE , et al.   Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability . Blood . 1998;92:1131–1141
  54. Dorrell C , Gan OI , Pereira DS , Hawlet RG , Dick JE . Expansion of human cord blood CD34+CD38− cells in ex vivo culture during retroviral transduction without a corresponding increase in SCID repopulating cell (SRC) frequency (dissociation of SRC phenotype and function) . Blood . 2000;95:102–110
  55. Gothot A , van der Loo JCM , Clapp DW , Srour EF . Cell cycle-related changes in repopulating capacity of human mobilized peripheral blood CD34+ cells in non-obese diabetic/ severe combined immune-deficient mice . Blood . 1998;92:2641–2649
  56. Luskey BD , Rosenblatt M , Zsebo K , Williams DA . Stem cell factor promotes retroviral-mediated gene transfer into murine hematopoietic stem cells . Blood . 1992;92:396–402
  57. Dao MA , Hannum CH , Kohn DB , Nolta JA . FLT3 ligand preserves the ability of human CD34+ progenitors to sustain long-term hematopoiesis in immune-deficient mice after ex vivo retroviral-mediated transduction . Blood . 1997;89:446–456
  58. Kiem HP , Andrews RG , Morris J , Peterson L , Heyward S , Allen JM , et al.   Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor . Blood . 1998;92:1878–1886
  59. Wu T , Kim HJ , Sellers SE , Meade KE , Agricola BA , Metzger ME , et al.   Prolonged high-level detection of retrovirally-marked hematopoietic cells in non-human primates after transduction of CD34+ progenitors using clinically feasible methods . Mol Ther . 2000;1:285–293
  60. Williams DA , Rios M , Stephens C , Patel VP . Fibronectin and VLA-4 in haematopoietic stem cell-microenvironment interactions . Nature . 1991;352:438–441
  61. Moritz T , Patel VP , Williams DA . Bone marrow extracellular matrix molecules improve gene transfer into human hematopoietic cells via retroviral vectors . J Clin Invest . 1994;93:1451–1457
  62. Hanenberg H , Xiao XL , Dilloo D , Hashino K , Kato I , Williams DA . Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells . Nat Med . 1996;2:876–882
  63. Yokota T , Oritani K , Mitsui H , Aoyema K , Ishikawa J , Sugahara H , et al.   Growth-supporting activities of fibronectin on hematopoietic stem/progenitor cells in vitro and in vivo (structural requirements for fibronectin activities of CS1 and cell-binding domains) . Blood . 1998;91:3263–3272
  64. Kotani H , Newton PB , Zhang S , Chiang YL , Otto E , Weaver L , et al.   Improved methods of retroviral vector transduction and production for gene therapy . Hum Gene Ther . 1994;5:19–28
  65. Chien ML , Foster JL , Douglas JL , Garcia JV . The amphotropic murine leukemia virus receptor gene encodes a 71-kilodalton protein that is induced by phosphate depletion . J Virol . 1997;71:4564–4570
  66. Orlic D , Girard LJ , Jordan CT , Anderson SM , Cline AP , Bodine DM . The level of mRNA encoding the amphotropic retrovirus receptor in mouse and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction . Proc Natl Acad Sci USA . 1996;93:11097–11102
  67. Olah Z , Lehel C , Anderson WB , Eiden MV , Wilson CA . The cellular receptor for gibbon ape leukemia virus is a novel high affinity sodium-dependent phosphate transporter . J Biol Chem . 1994;269:25426–25431
  68. Conneally E , Eaves CJ , Humphries RK . Efficient retroviral-mediated gene transfer to human cord blood stem cells with in vivo repopulating potential . Blood . 1998;91:3487–3493
  69. Hennemann B , Conneally E , Pawliuk R , Leboulch P , Rose-John S , Reid D , et al.   Optimization of retroviral-mediated gene transfer to human NOD/SCID mouse repopulating cord blood cells through a systematic analysis of protocol variables . Exp Hematol . 1999;27:817–825
  70. Schilz AJ , Brouns G , Knoss H , Ottmann OG , Hoelzer D , Fauser AA , et al.   High efficiency gene transfer to human hematopoietic SCID-repopulating cells under serum-free conditions . Blood . 1998;92:3163–3171
  71. Whitwam T , Haskins ME , Henthorn PS , Kraszewski JN , Kleiman SE , Seidel NE , et al.   Retroviral marking of canine bone marrow (long-term, high-level expression of human interleukin-2 receptor common gamma chain in canine lymphocytes) . Blood . 1998;92:1565–1575
  72. Kiem HP , Heyward S , Winkler A , Potter J , Allen JM , Miller AD , et al.   Gene transfer into marrow repopulating cells (Comparison between amphotropic and gibbon ape leukemia virus pseudotyped retroviral vectors in a competitive repopulation assay in baboons) . Blood . 1997;90:4638–4645
  73. Burns JC , Friedmann T , Driever W , Burrascano M , Yee JK . Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors (Concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells) . Proc Natl Acad Sci USA . 1993;90:8033–8037
  74. von Laer D , Corovic A , Vogt B , Herwig U , Roscher S , Fehse B , et al.   Amphotropic and VSV-G-pseudotyped retroviral vectors transduce human hematopoietic progenitor cells with similar efficiency . Bone Marrow Transplant . 2000;25(suppl 2):S75–S79
  75. Depolo NJ , Reed JD , Sheridan PL , Townsend K , Sauter SL , Jolly DJ , et al.   VSV-G pseudotyped lentiviral vector particles produced in human cells are inactivated by human serum . Mol Ther . 2000;2:218–222
  76. Cosset FL , Takeuchi Y , Battini JL , Weiss RA , Collins MK . High-titer packaging cells producing recombinant retroviruses resistant to human serum . J Virol . 1995;69:7430–7436
  77. Hanawa H , Kelly PF , Nathwani AC , Persons DA , Vandergriff J , Hargrove P , et al.   Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood . Mol Ther . 2002;5:242–251
  78. Sandrin V , Boson B , Salmon P , Gay W , Negre D , Le Grand R , et al.   Lentiviral vectors pseudotyped with a modified RD114 envelope glycoprotein show increased stability in sera and augmented transduction of primary lymphocytes and CD34+ cells derived from human and nonhuman primates . Blood . 2002;100:823–832
  79. Rasko JE , Battini JL , Gottschalk RJ , Mazo I , Miller AD . The RD114/simian type D retrovirus receptor is a neutral amino acid transporter . Proc Natl Acad Sci USA . 1999;96:2129–2134
  80. Goerner M , Horn PA , Peterson L , Kurre P , Storb R , Rasko JE , et al.   Sustained multilineage gene persistence and expression in dogs transplanted with CD34(+) marrow cells transduced by RD114-pseudotype oncoretrovirus vectors . Blood . 2001;98:2065–2070
  81. Brenner S , Whiting-Theobald NL , Linton GF , Holmes KL , Anderson-Cohen M , Kelly PF , et al.   Concentrated RD114-pseudotyped MFGS-gp91phox vector achieves high levels of functional correction of the chronic granulomatous disease oxidase defect in NOD/SCID/beta-microglobulin-/- repopulating mobilized human peripheral blood CD34+ cells . Blood . 2003;102:2789–2797
  82. Gatlin J , Melkus MW , Padgett A , Kelly PF , Garcia JV . Engraftment of NOD/SCID mice with human CD34(+) cells transduced by concentrated oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein . J Virol . 2001;75:9995–9999
  83. Kelly PF , Vandergriff J , Nathwani A , Nienhuis AW , Vanin EF . Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein . Blood . 2000;96:1206–1214
  84. Chan SY , Speck RF , Ma MC , Goldsmith MA . Distinct mechanisms of entry by envelope glycoproteins of Marburg and Ebola (Zaire) viruses . J Virol . 2000;74:4933–4937
  85. Lewis BC , Chinnasamy N , Morgan RA , Varmus HE . Development of an avian leukosis-sarcoma virus subgroup A pseudotyped lentiviral vector . J Virol . 2001;75:9339–9344
  86. Larochelle A , Peng KW , Russell SJ . Lentiviral vector targeting . Curr Top Microbiol Immunol . 2002;261:143–163
  87. An DS , Wersto RP , Agricola BA , Metzger M , Lu S , Amado RG , et al.   Marking and gene expression by a lentivirus vector in transplanted human and nonhuman primate CD34+ cells . J Virol . 2000;74:1286–1295
  88. An DS , Kung SK , Bonifacino A , Wersto RP , Metzger ME , Agricola BA , et al.   Lentivirus vector-mediated hematopoietic stem cell gene transfer of common gamma-chain cytokine receptor in rhesus macaques . J Virol . 2001;75:3547–3555
  89. Horn PA , Morris JC , Bukovsky AA , Andrews RG , Naldini L , Kurre P , et al.   Lentivirus-mediated gene transfer into hematopoietic repopulating cells in baboons . Gene Ther . 2002;9:1464–1471
  90. Hanawa H , Hematti P , Keyvanfar K , Metzger ME , Krouse A , Donahue RE , et al.   Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system . Blood . 2004;103:4062–4069
  91. Case SS , Price MA , Jordan CT , Yu XJ , Wang L , Bauer G , et al.   Stable transduction of quiescent CD34+CD38− human hematopoietic cells by HIV-1-based lentiviral vectors . Proc Natl Acad Sci USA . 1999;96:2988–2993
  92. Uchida N , Sutton RE , Friera AM , He D , Reitsma MJ , Chang WC , et al.   HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated Go/G1 human hematopoietic stem cells . Proc Natl Acad Sci USA . 1998;95:11939–11944
  93. Miyoshi H , Smith KA , Mosier DE , Verma IM , Torbett BE . Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors . Science . 1999;283:682–686
  94. Piacibello W , Bruno S , Sanavio F , Droetto S , Gunetti M , Ailles L , 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 . Blood . 2002;100:4391–4400
  95. Miyoshi H , Smith KA , Mosier DE , Verma IM , Torbett BE . Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors . Science . 1999;283:682–686
  96. Woods NB , Fahlman C , Mikkola H , Hamaguchi I , Olsson K , Zufferey R , et al.   Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells . Blood . 2000;96:3725–3733
  97. Sutton RE , Reitsma MJ , Uchida N , Brown PO . Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell cycle dependent . J Virol . 1999;73:3649–3660
  98. Takatoku M , Sellers S , Agricola BA , Metzger ME , Kato I , Donahue RE , et al.   Avoidance of stimulation improves engraftment of cultured and retrovirally transduced hematopoietic cells in primates . J Clin Invest . 2001;108:447–455
  99. Kiem HP , Rasko JE , Morris J , Peterson L , Kurre P , Andrews RG . Ex vivo selection for oncoretrovirally transduced green fluorescent protein-expressing CD34-enriched cells increases short-term engraftment of transduced cells in baboons . Hum Gene Ther . 2002;13:891–899
  100. Bonini C , Ferrari G , Verzletti S , Servida P , Zappone E , Ruggieri L , et al.   HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia . Science . 1997;276:1719–1724
  101. Li Z , Dullmann J , Schiedlmeier B , Schmidt M , von Kalle C , Meyer J , et al.   Murine leukemia induced by retroviral gene marking . Science . 2002;296:497
  102. Bonini C , Grez M , Traversari C , Ciceri F , Marktel S , Ferrari G , et al.   Safety of retroviral gene marking with a truncated NGF receptor . Nat Med . 2003;9:367–369
  103. Sorrentino BP , Brandt SJ , Bodine D , Gottesman M , Pastan I , Cline A , et al.   Selection of drug resistant bone marrow cells in vivo after retroviral transfer of human MDR1 . Science . 1992;257:99–103
  104. Abonour R , Williams DA , Einhorn L , Hall KM , Chen J , Coffman J , et al.   Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells . Nat Med . 2000;6:652–658
  105. Cowan KH , Moscow JA , Huang H , Zujewski J , O’Shaughnessy J , Sorrentino B , et al.   Paclitaxel chemotherapy following autologous stem cell transplantation and engraftment of hematopoietic cells transduced with a retrovirus containing the multidrug resistance cDNA (MDR1) in metastatic breast cancer patients . Clin Cancer Res . 1999;5:1619–1628
  106. Moscow JA , Huang H , Sorrentino B , Zujewski J , Chiang Y , Chang YN , et al.   Effect of MDR1 gene transduction on engraftment and expansion of hematopoietic progenitor cells during breast cancer chemotherapy . Blood . 1999;94:52–61
  107. Bunting KD , Galipeau J , Topham D , Benaim E , Sorrentino BP . Transduction of murine bone marrow cells with an MDR1 vector enables ex vivo stem cell expansion, but these expanded grafts cause a myeloproliferative syndrome in transplanted mice . Blood . 1998;92:2269–2279
  108. Sellers SE , Tisdale JF , Agricola BA , Metzger ME , Donahue RE , Dunbar CE , et al.   The effect of multidrug-resistance 1 gene versus neo transduction on ex vivo and in vivo expansion of rhesus macaque hematopoietic repopulating cells . Blood . 2001;97:1888–1891
  109. Allay JA , Persons DA , Galipeau D , Riberdy JM , Ashmun RA , Blakely RL , et al.   In vivo selection of retrovirally transduced hematopoietic stem cells . Nat Med . 1998;4:1136–1143
  110. Corey CA , DeSilva AD , Holland CA , Williams DA . Serial transplantation of methotrexate-resistant bone marrow (Protection of murine recipeints from drug toxicity by progeny of transduced stem cells) . Blood . 1990;75:337–343
  111. Persons DA , Allay JA , Bonifacino A , Lu T , Agricola B , Metzger ME , et al.   Transient in vivo selection of transduced peripheral blood cells using antifolate drug selection in rhesus macaques that received transplants with hematopoietic stem cells expressing dihydrofolate reductase vectors . Blood . 2004;103:796–803
  112. Davis BM , Reese JS , Koc ON , Lee K , Schupp JE , Gerson SL . Selection for G156A O6-methylguanine DNA methyltransferase gene-transduced hematopoietic progenitors and protection from lethality in mice treated with O6-benzylguanine and 1,3-bis(2-chloroethyl)-1- nitrosourea . Cancer Res . 1997;57:5093–5099
  113. Davis BM , Koc ON , Gerson SL . Limiting numbers of G156A O(6)-methylguanine-DNA methyltransferase-transduced marrow progenitors repopulate nonmyeloablated mice after drug selection . Blood . 2000;95:3078–3084
  114. Persons DA , Allay ER , Sawai N , Hargrove PW , Brent TP , Hanawa H , et al.   Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells . Blood . 2003;102:506–513
  115. Neff T , Horn PA , Peterson LJ , Thomasson BM , Thompson J , Williams DA , et al.   Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model . J Clin Invest . 2003;112:1581–1588
  116. Zielske SP , Reese JS , Lingas KT , Donze JR , Gerson SL . In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning . J Clin Invest . 2003;112:1561–1570
  117. Ito K , Ueda Y , Kokubun M , Urabe M , Inaba T , Mano H , et al.   Development of a novel selective amplifier gene for controllable expansion of transduced hematopoietic cells . Blood . 1997;90:3884–3892
  118. Hanazono Y , Nagashima T , Takatoku M , Shibata H , Ageyama N , Asano T , et al.   In vivo selective expansion of gene-modified hematopoietic cells in a nonhuman primate model . Gene Ther . 2002;9:1055–1064
  119. Blau CA , Peterson KR , Drachman JG , Spencer DM . A proliferation switch for genetically modified cells . Proc Natl Acad Sci USA . 1997;94:3076–3081
  120. Jin L , Siritanaratkul N , Emery DW , Richard RE , Kaushansky K , Papayannopoulou T , et al.   Targeted expansion of genetically modified bone marrow cells . Proc Natl Acad Sci USA . 1998;95:8093–8097
  121. Antonchuk J , Sauvageau G , Humphries RK . HOXB4-induced expansion of adult hematopoietic stem cells ex vivo . Cell . 2002;109:39–45
  122. Schiedlmeier B , Klump H , Will E , Arman-Kalcek G , Li Z , Wang Z , et al.   High-level ectopic HOXB4 expression confers a profound in vivo competitive growth advantage on human cord blood CD34+ cells, but impairs lymphomyeloid differentiation . Blood . 2003;101:1759–1768
  123. Stier S , Cheng T , Forkert R , Lutz C , Dombkowski DM , Zhang JL , et al.   Ex vivo targeting of p21Cip1/Waf1 permits relative expansion of human hematopoietic stem cells . Blood . 2003;102:1260–1266

PII: S0037-1963(04)00125-8

doi: 10.1053/j.seminhematol.2004.07.002

Seminars in Hematology
Volume 41, Issue 4 , Pages 257-271 , October 2004

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