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Volumn 107, Issue 5, 2006, Pages 1751-1760

Survival of the fittest: In vivo selection and stem cell gene therapy

Author keywords

[No Author keywords available]

Indexed keywords

6 O METHYLGUANINE; ADENOSINE DEAMINASE; BUSULFAN; CARMUSTINE; CYCLOPHOSPHAMIDE; DIHYDROFOLATE REDUCTASE; ESTROGEN; ETOPOSIDE; IRON; JANUS KINASE 3; METHYLGUANINE METHYLTRANSFERASE; MULTIDRUG RESISTANCE PROTEIN 1; PACLITAXEL; POLYDEOXYRIBONUCLEOTIDE SYNTHASE; PROTEIN KINASE ZAP 70; RAG2 PROTEIN; TAMOXIFEN; TEMOZOLOMIDE; TRIMETREXATE; UNCLASSIFIED DRUG; WISKOTT ALDRICH SYNDROME PROTEIN;

EID: 33344476395     PISSN: 00064971     EISSN: 00064971     Source Type: Journal    
DOI: 10.1182/blood-2005-06-2335     Document Type: Review
Times cited : (53)

References (121)
  • 1
    • 0037189401 scopus 로고    scopus 로고
    • Correction of ADASCID by stem cell gene therapy combined with nonmyeloablative conditioning
    • Aiuti A, Slavin S, Aker M, et al. Correction of ADASCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science. 2002;296:2410-2413.
    • (2002) Science , vol.296 , pp. 2410-2413
    • Aiuti, A.1    Slavin, S.2    Aker, M.3
  • 2
    • 19944421384 scopus 로고    scopus 로고
    • Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
    • Gaspar HB, Parsley KL, Howe S, et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet. 2004;364:2181-2187.
    • (2004) Lancet , vol.364 , pp. 2181-2187
    • Gaspar, H.B.1    Parsley, K.L.2    Howe, S.3
  • 3
    • 0037129435 scopus 로고    scopus 로고
    • Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
    • 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;346:1185-1193.
    • (2002) N Engl J Med , vol.346 , pp. 1185-1193
    • Hacein-Bey-Abina, S.1    Le Deist, F.2    Carlier, F.3
  • 4
    • 0034724857 scopus 로고    scopus 로고
    • Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
    • Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 2000;288:669-672.
    • (2000) Science , vol.288 , pp. 669-672
    • Cavazzana-Calvo, M.1    Hacein-Bey, S.2    De Saint Basile, G.3
  • 5
    • 0036892648 scopus 로고    scopus 로고
    • Highly efficient gene transfer into baboon marrow repopulating cells using GALV-pseudotype oncoretroviral vectors produced by human packaging cells
    • Horn PA, Topp MS, Morris JC, Riddell SR, Kiem H-P. Highly efficient gene transfer into baboon marrow repopulating cells using GALV-pseudotype oncoretroviral vectors produced by human packaging cells. Blood. 2002;100:3960-3967.
    • (2002) Blood , vol.100 , pp. 3960-3967
    • Horn, P.A.1    Topp, M.S.2    Morris, J.C.3    Riddell, S.R.4    Kiem, H.-P.5
  • 6
    • 0034153385 scopus 로고    scopus 로고
    • Prolonged high-level detection of retrovirally marked hematopoietic cells in nonhuman primates after transduction of CD34+ progenitors using clinically feasible methods
    • Wu T, Kim HJ, Sellers SE, et al. Prolonged high-level detection of retrovirally marked hematopoietic cells in nonhuman primates after transduction of CD34+ progenitors using clinically feasible methods. Mol Ther. 2000;1:285-293.
    • (2000) Mol Ther , vol.1 , pp. 285-293
    • Wu, T.1    Kim, H.J.2    Sellers, S.E.3
  • 7
    • 1342332882 scopus 로고    scopus 로고
    • Efficient gene transfer to hematopoietic repopulating cells using concentrated RD114-pseudotype vectors produced by human packaging cells
    • Neff T, Peterson LJ, Morris JC, et al. Efficient gene transfer to hematopoietic repopulating cells using concentrated RD114-pseudotype vectors produced by human packaging cells [letter]. Mol Ther. 2004;9:157-159.
    • (2004) Mol Ther , vol.9 , pp. 157-159
    • Neff, T.1    Peterson, L.J.2    Morris, J.C.3
  • 8
    • 0029859412 scopus 로고    scopus 로고
    • 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
    • U S A
    • Dunbar CE, Seidel NE, Doren S, 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 U S A. 1996;93:11871-11876.
    • (1996) Proc Natl Acad Sci , vol.93 , pp. 11871-11876
    • Dunbar, C.E.1    Seidel, N.E.2    Doren, S.3
  • 9
    • 0025089990 scopus 로고
    • Serial transplantation of methotrexate-resistant bone marrow: Protection of murine recipients from drug toxicity by progeny of transduced stem cells
    • Corey CA, DeSilva AD, Holland CA, Williams DA. Serial transplantation of methotrexate-resistant bone marrow: protection of murine recipients from drug toxicity by progeny of transduced stem cells. Blood. 1990;75:337-343.
    • (1990) Blood , vol.75 , pp. 337-343
    • Corey, C.A.1    DeSilva, A.D.2    Holland, C.A.3    Williams, D.A.4
  • 10
    • 0023925750 scopus 로고
    • Canine model for gene therapy: Inefficient gene expression in dogs reconstituted with autologous marrow infected with retroviral vectors
    • 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.
    • (1988) Blood , vol.71 , pp. 742-747
    • Stead, R.B.1    Kwok, W.W.2    Storb, R.3    Miller, A.D.4
  • 11
    • 0031593688 scopus 로고    scopus 로고
    • T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates
    • Kohn DB, Hershfield MS, Carbonaro D, et al. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nat Med. 1998;4:775-780.
    • (1998) Nat Med , vol.4 , pp. 775-780
    • Kohn, D.B.1    Hershfield, M.S.2    Carbonaro, D.3
  • 12
    • 0347086142 scopus 로고    scopus 로고
    • Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model
    • Neff T, Horn PA, Peterson LJ, 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.
    • (2003) J Clin Invest , vol.112 , pp. 1581-1588
    • Neff, T.1    Horn, P.A.2    Peterson, L.J.3
  • 13
    • 1642541226 scopus 로고    scopus 로고
    • 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
    • Persons DA, Allay DA, Bonifacino A, 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.
    • (2004) Blood , vol.103 , pp. 796-803
    • Persons, D.A.1    Allay, D.A.2    Bonifacino, A.3
  • 14
    • 0037022610 scopus 로고    scopus 로고
    • Drug selection with paclitaxel restores expression of linked IL-2 receptor gamma-chain and multidrug resistance (MDR1) transgenes in canine bone marrow
    • U S A
    • Licht T, Haskins M, Henthorn P, et al. Drug selection with paclitaxel restores expression of linked IL-2 receptor gamma-chain and multidrug resistance (MDR1) transgenes in canine bone marrow. Proc Natl Acad Sci U S A. 2002;99:3123-3128.
    • (2002) Proc Natl Acad Sci , vol.99 , pp. 3123-3128
    • Licht, T.1    Haskins, M.2    Henthorn, P.3
  • 15
    • 0028789792 scopus 로고
    • Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients
    • Bordignon C, Notarangelo LD, Nobili N, et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science. 1995;270:470-475.
    • (1995) Science , vol.270 , pp. 470-475
    • Bordignon, C.1    Notarangelo, L.D.2    Nobili, N.3
  • 16
    • 0034694076 scopus 로고    scopus 로고
    • Self-selection by genetically modified committed lymphocyte precursors reverses the phenotype of JAK3-deficient mice without myeloablation
    • Bunting KD, Lu T, Kelly PF, Sorrentino BP. Self-selection by genetically modified committed lymphocyte precursors reverses the phenotype of JAK3-deficient mice without myeloablation. Hum Gene Ther. 2000;11:2353-2364.
    • (2000) Hum Gene Ther , vol.11 , pp. 2353-2364
    • Bunting, K.D.1    Lu, T.2    Kelly, P.F.3    Sorrentino, B.P.4
  • 17
    • 0031890632 scopus 로고    scopus 로고
    • Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer
    • Bunting KD, Sangster MY, Ihle JN, Sorrentino BP. Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer. Nat Med. 1998;4:58-64.
    • (1998) Nat Med , vol.4 , pp. 58-64
    • Bunting, K.D.1    Sangster, M.Y.2    Ihle, J.N.3    Sorrentino, B.P.4
  • 18
    • 0036891848 scopus 로고    scopus 로고
    • Gene therapy of RAG-2-/- Mice: Sustained correction of the immunodeficiency
    • Yates F, Malassis-Seris M, Stockholm D, et al. Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency. Blood. 2002;100:3942-3949.
    • (2002) Blood , vol.100 , pp. 3942-3949
    • Yates, F.1    Malassis-Seris, M.2    Stockholm, D.3
  • 19
    • 0037103385 scopus 로고    scopus 로고
    • Reconstitution of lymphoid development and function in ZAP-70-deficient mice following gene transfer into bone marrow cells
    • Otsu M, Steinberg M, Ferrand C, et al. Reconstitution of lymphoid development and function in ZAP-70-deficient mice following gene transfer into bone marrow cells. Blood. 2002;100:1248-1256.
    • (2002) Blood , vol.100 , pp. 1248-1256
    • Otsu, M.1    Steinberg, M.2    Ferrand, C.3
  • 20
    • 0037443395 scopus 로고    scopus 로고
    • Gene therapy for Wiskott-Aldrich syndrome: Rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice
    • Klein C, Nguyen D, Liu CH, et al. Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice. Blood. 2003;101:2159-2166.
    • (2003) Blood , vol.101 , pp. 2159-2166
    • Klein, C.1    Nguyen, D.2    Liu, C.H.3
  • 21
    • 0141601975 scopus 로고    scopus 로고
    • Defects in T-cell-mediated immunity to influenza virus in murine Wiskott-Aldrich syndrome are corrected by oncoretroviral vector-mediated gene transfer into repopulating hematopoietic cells
    • Strom TS, Turner SJ, Andreansky S, et al. Defects in T-cell-mediated immunity to influenza virus in murine Wiskott-Aldrich syndrome are corrected by oncoretroviral vector-mediated gene transfer into repopulating hematopoietic cells. Blood. 2003;102:3108-3116.
    • (2003) Blood , vol.102 , pp. 3108-3116
    • Strom, T.S.1    Turner, S.J.2    Andreansky, S.3
  • 22
    • 0035902552 scopus 로고    scopus 로고
    • Somatic mosaicism in Wiskott-Aldrich syndrome suggests in vivo reversion by a DNA slippage mechanism
    • U S A
    • Wada T, Schurman SH, Otsu M, et al. Somatic mosaicism in Wiskott-Aldrich syndrome suggests in vivo reversion by a DNA slippage mechanism. Proc Natl Acad Sci U S A. 2001;98:8697-8702.
    • (2001) Proc Natl Acad Sci , vol.98 , pp. 8697-8702
    • Wada, T.1    Schurman, S.H.2    Otsu, M.3
  • 23
    • 0043240225 scopus 로고    scopus 로고
    • A defect in hematopoietic stem cell migration explains the nonrandom X-chromosome inactivation in carriers of Wiskott-Aldrich syndrome
    • Lacout C, Haddad E, Sabri S, et al. A defect in hematopoietic stem cell migration explains the nonrandom X-chromosome inactivation in carriers of Wiskott-Aldrich syndrome. Blood. 2003;102:1282-1289.
    • (2003) Blood , vol.102 , pp. 1282-1289
    • Lacout, C.1    Haddad, E.2    Sabri, S.3
  • 24
    • 25144449181 scopus 로고    scopus 로고
    • A human ortholog of archaeal DNA repair protein Hef is defective in Fanconi anemia complementation group M
    • Meetei AR, Medhurst AL, Ling C, et al. A human ortholog of archaeal DNA repair protein Hef is defective in Fanconi anemia complementation group M. Nat Genet. 2005;37:958-963.
    • (2005) Nat Genet , vol.37 , pp. 958-963
    • Meetei, A.R.1    Medhurst, A.L.2    Ling, C.3
  • 25
    • 25144457604 scopus 로고    scopus 로고
    • The DNA helicase BRIP1 is defective in Fanconi anemia complementation group J
    • Levitus M, Waisfisz Q, Godthelp BC, et al. The DNA helicase BRIP1 is defective in Fanconi anemia complementation group J. Nat Genet. 2005;37:934-935.
    • (2005) Nat Genet. , vol.37 , pp. 934-935
    • Levitus, M.1    Waisfisz, Q.2    Godthelp, B.C.3
  • 26
    • 0037268338 scopus 로고    scopus 로고
    • The Fanconi anaemia/BRCA pathway
    • D'Andrea AD, Grompe M. The Fanconi anaemia/BRCA pathway (Review). Nat Rev Cancer. 2003;3:23-34.
    • (2003) Nat Rev Cancer , vol.3 , pp. 23-34
    • D'Andrea, A.D.1    Grompe, M.2
  • 27
    • 13344278020 scopus 로고    scopus 로고
    • Inactivation of Fac in mice produces inducible chromosomal instability and reduced fertility reminiscent of Fanconi anaemia
    • Chen M, Tomkins DJ, Auerbach W, et al. Inactivation of Fac in mice produces inducible chromosomal instability and reduced fertility reminiscent of Fanconi anaemia. Nat Genet. 1996;12:448-451.
    • (1996) Nat Genet , vol.12 , pp. 448-451
    • Chen, M.1    Tomkins, D.J.2    Auerbach, W.3
  • 28
    • 0033852602 scopus 로고    scopus 로고
    • Mice with a targeted disruption of the Fanconi anemia homolog Fanca
    • Cheng NC, van de Vrugt HJ, van der Valk MA, et al. Mice with a targeted disruption of the Fanconi anemia homolog Fanca. Hum Mol Genet. 2000;9:1805-1811.
    • (2000) Hum Mol Genet , vol.9 , pp. 1805-1811
    • Cheng, N.C.1    Van De Vrugt, H.J.2    Van Der Valk, M.A.3
  • 29
    • 0042519602 scopus 로고    scopus 로고
    • Epithelial cancer in Fanconi anemia complementation group D2 (Fancd2) knockout mice
    • Houghtaling S, Timmers C, Noll M, et al. Epithelial cancer in Fanconi anemia complementation group D2 (Fancd2) knockout mice. Genes Dev. 2003;17:2021-2035.
    • (2003) Genes Dev , vol.17 , pp. 2021-2035
    • Houghtaling, S.1    Timmers, C.2    Noll, M.3
  • 30
    • 0035760285 scopus 로고    scopus 로고
    • Targeted disruption of the murine Fanconi anemia gene, Fancg/Xrcc9
    • Yang Y, Kuang Y, De Oca RM, et al. Targeted disruption of the murine Fanconi anemia gene, Fancg/Xrcc9. Blood. 2001;98:3435-3440.
    • (2001) Blood , vol.98 , pp. 3435-3440
    • Yang, Y.1    Kuang, Y.2    De Oca, R.M.3
  • 31
    • 0034650931 scopus 로고    scopus 로고
    • Phenotypic correction of Fanconi anemia group C knockout mice
    • Gush KA, Fu KL, Grompe M, Walsh CE. Phenotypic correction of Fanconi anemia group C knockout mice. Blood. 2000;95:700-704.
    • (2000) Blood , vol.95 , pp. 700-704
    • Gush, K.A.1    Fu, K.L.2    Grompe, M.3    Walsh, C.E.4
  • 32
    • 0034981121 scopus 로고    scopus 로고
    • Preclinical protocol for in vivo selection of hematopoietic stem cells corrected by gene therapy in Fanconi anemia group C
    • Noll M, Bateman RL, D'Andrea AD, Grompe M. Preclinical protocol for in vivo selection of hematopoietic stem cells corrected by gene therapy in Fanconi anemia group C. Mol Ther. 2001;3:14-23.
    • (2001) Mol Ther , vol.3 , pp. 14-23
    • Noll, M.1    Bateman, R.L.2    D'Andrea, A.D.3    Grompe, M.4
  • 33
    • 0142150968 scopus 로고    scopus 로고
    • Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector
    • Yamada K, Ramezani A, Hawley RG, et al. Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector. Mol Ther. 2003;8:600-610.
    • (2003) Mol Ther , vol.8 , pp. 600-610
    • Yamada, K.1    Ramezani, A.2    Hawley, R.G.3
  • 34
    • 0037441890 scopus 로고    scopus 로고
    • Retroviral-mediated expression of recombinant Fancc enhances the repopulating ability of Fancc-/- hematopoietic stem cells and decreases the risk of clonal evolution
    • Haneline LS, Li X, Ciccone SL, et al. Retroviral-mediated expression of recombinant Fancc enhances the repopulating ability of Fancc-/- hematopoietic stem cells and decreases the risk of clonal evolution. Blood. 2003;101:1299-1307.
    • (2003) Blood , vol.101 , pp. 1299-1307
    • Haneline, L.S.1    Li, X.2    Ciccone, S.L.3
  • 35
    • 0032796554 scopus 로고    scopus 로고
    • Spontaneous functional correction of homozygous Fanconi anaemia alleles reveals novel mechanistic basis for reverse mosaicism
    • Waisfisz Q, Morgan NV, Savino M, et al. Spontaneous functional correction of homozygous Fanconi anaemia alleles reveals novel mechanistic basis for reverse mosaicism. Nat Genet. 1999;22:379-383.
    • (1999) Nat Genet , vol.22 , pp. 379-383
    • Waisfisz, Q.1    Morgan, N.V.2    Savino, M.3
  • 36
    • 0035956959 scopus 로고    scopus 로고
    • Somatic mosaicism in Fanconi anemia: Evidence of genotypic reversion in lymphohematopoietic stem cells
    • U S A
    • Gregory JJ Jr, Wagner JE, Verlander PC, et al. Somatic mosaicism in Fanconi anemia: evidence of genotypic reversion in lymphohematopoietic stem cells. Proc Natl Acad Sci U S A. 2001;98:2532-2537.
    • (2001) Proc Natl Acad Sci , vol.98 , pp. 2532-2537
    • Gregory Jr., J.J.1    Wagner, J.E.2    Verlander, P.C.3
  • 37
    • 0034115968 scopus 로고    scopus 로고
    • Haematopoietic cell transplantation in patients with Fanconi anaemia using alternate donors: Results of a total body irradiation dose escalation trial
    • MacMillan ML, Auerbach AD, Davies SM, et al. Haematopoietic cell transplantation in patients with Fanconi anaemia using alternate donors: results of a total body irradiation dose escalation trial. Br J Haematol. 2000;109:121-129.
    • (2000) Br J Haematol , vol.109 , pp. 121-129
    • MacMillan, M.L.1    Auerbach, A.D.2    Davies, S.M.3
  • 38
    • 12944315003 scopus 로고    scopus 로고
    • Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: A study on behalf of the European Group for Blood and Marrow Transplantation
    • Guardiola P, Pasquini R, Dokal I, et al. Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation. Blood. 2000;95:422-429.
    • (2000) Blood , vol.95 , pp. 422-429
    • Guardiola, P.1    Pasquini, R.2    Dokal, I.3
  • 40
    • 0141484484 scopus 로고    scopus 로고
    • Molecular assessment of erythroid lineage chimerism following nonmyeloablative allogeneic stem cell transplantation
    • Wu CJ, Hochberg EP, Rogers SA, et al. Molecular assessment of erythroid lineage chimerism following nonmyeloablative allogeneic stem cell transplantation. Exp Hematol. 2003;31:924-933.
    • (2003) Exp Hematol , vol.31 , pp. 924-933
    • Wu, C.J.1    Hochberg, E.P.2    Rogers, S.A.3
  • 41
    • 0027214314 scopus 로고
    • Correction of murine beta-thalassemia by partial bone marrow chimerism: Selective advantage of normal erythropoiesis
    • van den Bos C, Kieboom D, Wagemaker G. Correction of murine beta-thalassemia by partial bone marrow chimerism: selective advantage of normal erythropoiesis. Bone Marrow Transplant. 1993;12:9-13.
    • (1993) Bone Marrow Transplant , vol.12 , pp. 9-13
    • Van Den Bos, C.1    Kieboom, D.2    Wagemaker, G.3
  • 42
    • 0035874519 scopus 로고    scopus 로고
    • Functional requirements for phenotypic correction of murine beta-thalassemia: Implications for human gene therapy
    • Persons DA, Allay ER, Sabatino DE, Kelly P, Bodine DM, Nienhuis AW. Functional requirements for phenotypic correction of murine beta-thalassemia: implications for human gene therapy. Blood. 2001;97:3275-3282.
    • (2001) Blood , vol.97 , pp. 3275-3282
    • Persons, D.A.1    Allay, E.R.2    Sabatino, D.E.3    Kelly, P.4    Bodine, D.M.5    Nienhuis, A.W.6
  • 43
    • 10744229369 scopus 로고    scopus 로고
    • Chimerism and cure: Hematologic and pathologic correction of murine sickle cell disease
    • Kean LS, Manci EA, Perry J, et al. Chimerism and cure: hematologic and pathologic correction of murine sickle cell disease. Blood. 2003;102:4582-4593.
    • (2003) Blood , vol.102 , pp. 4582-4593
    • Kean, L.S.1    Manci, E.A.2    Perry, J.3
  • 44
    • 0030848104 scopus 로고    scopus 로고
    • Phlebotomy to reduce iron overload in patients cured of thalassemia by bone marrow transplantation: Italian Cooperative Group for Phlebotomy Treatment of Transplanted Thalassemia Patients
    • Angelucci E, Muretto P, Lucarelli G, et al. Phlebotomy to reduce iron overload in patients cured of thalassemia by bone marrow transplantation: Italian Cooperative Group for Phlebotomy Treatment of Transplanted Thalassemia Patients. Blood. 1997;90:994-998.
    • (1997) Blood , vol.90 , pp. 994-998
    • Angelucci, E.1    Muretto, P.2    Lucarelli, G.3
  • 45
    • 0019792931 scopus 로고
    • Iron overload and desferrioxamine chelation therapy in beta-thalassemia intermedia
    • Cossu P, Toccafondi C, Vardeu F, et al. Iron overload and desferrioxamine chelation therapy in beta-thalassemia intermedia. Eur J Pediatr. 1981;137:267-271.
    • (1981) Eur J Pediatr , vol.137 , pp. 267-271
    • Cossu, P.1    Toccafondi, C.2    Vardeu, F.3
  • 47
    • 0346433908 scopus 로고    scopus 로고
    • Decreased hepcidin mRNA expression in thalassemic mice
    • Adamsky K, Weizer O, Amariglio N, et al. Decreased hepcidin mRNA expression in thalassemic mice. Br J Haematol. 2004;124:123-124.
    • (2004) Br J Haematol , vol.124 , pp. 123-124
    • Adamsky, K.1    Weizer, O.2    Amariglio, N.3
  • 49
    • 0037772187 scopus 로고    scopus 로고
    • Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells
    • Persons DA, Allay ER, Sawai N, 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.
    • (2003) Blood , vol.102 , pp. 506-513
    • Persons, D.A.1    Allay, E.R.2    Sawai, N.3
  • 50
    • 0035353166 scopus 로고    scopus 로고
    • Pharmacologically regulated cell therapy
    • Neff T, Blau CA. Pharmacologically regulated cell therapy (Review). Blood. 2001;97:2535-2540.
    • (2001) Blood , vol.97 , pp. 2535-2540
    • Neff, T.1    Blau, C.A.2
  • 51
    • 0037105534 scopus 로고    scopus 로고
    • Pharmacologically regulated in vivo selection in a large animal
    • Neff T, Horn PA, Valli VE, et al. Pharmacologically regulated in vivo selection in a large animal. Blood. 2002;100:2026-2031.
    • (2002) Blood , vol.100 , pp. 2026-2031
    • Neff, T.1    Horn, P.A.2    Valli, V.E.3
  • 52
    • 0033135855 scopus 로고    scopus 로고
    • The common marmoset as a target preclinical primate model for cytokine and gene therapy studies
    • Hibino H, Tani K, Ikebuchi K, et al. The common marmoset as a target preclinical primate model for cytokine and gene therapy studies. Blood. 1999;93:2839-2848.
    • (1999) Blood , vol.93 , pp. 2839-2848
    • Hibino, H.1    Tani, K.2    Ikebuchi, K.3
  • 53
    • 0033168346 scopus 로고    scopus 로고
    • Engraftment of MDR1 and NeoR gene-transduced hematopoietic cells after breast cancer chemotherapy
    • Moscow JA, Huang H, Carter C, et al. Engraftment of MDR1 and NeoR gene-transduced hematopoietic cells after breast cancer chemotherapy. Blood. 1999;94:52-61.
    • (1999) Blood , vol.94 , pp. 52-61
    • Moscow, J.A.1    Huang, H.2    Carter, C.3
  • 54
    • 0034131481 scopus 로고    scopus 로고
    • Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells
    • Abonour R, Williams DA, Einhorn L, 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.
    • (2000) Nat Med , vol.6 , pp. 652-658
    • Abonour, R.1    Williams, D.A.2    Einhorn, L.3
  • 55
    • 12844249961 scopus 로고    scopus 로고
    • Polyclonal chemoprotection against temozolomide in a large-animal model of drug resistance gene therapy
    • Neff T, Beard BC, Peterson LJ, Anandakumar P, Thompson J, Kiem H-P. Polyclonal chemoprotection against temozolomide in a large-animal model of drug resistance gene therapy. Blood. 2005;105:997-1002.
    • (2005) Blood , vol.105 , pp. 997-1002
    • Neff, T.1    Beard, B.C.2    Peterson, L.J.3    Anandakumar, P.4    Thompson, J.5    Kiem, H.-P.6
  • 56
    • 0036056412 scopus 로고    scopus 로고
    • In vivo selective expansion of gene-modified hematopoietic cells in a nonhuman primate model
    • Hanazono Y, Nagashima T, Takatoku M, et al. In vivo selective expansion of gene-modified hematopoietic cells in a nonhuman primate model. Gene Ther. 2002;9:1055-1064.
    • (2002) Gene Ther , vol.9 , pp. 1055-1064
    • Hanazono, Y.1    Nagashima, T.2    Takatoku, M.3
  • 57
    • 0033517123 scopus 로고    scopus 로고
    • STAT3 activation is sufficient to maintain an undifferentiated state of mouse embryonic stem cells
    • Matsuda T, Nakamura T, Nakao K, et al. STAT3 activation is sufficient to maintain an undifferentiated state of mouse embryonic stem cells. EMBO J. 1999;18:4261-4269.
    • (1999) EMBO J , vol.18 , pp. 4261-4269
    • Matsuda, T.1    Nakamura, T.2    Nakao, K.3
  • 58
    • 33344458419 scopus 로고    scopus 로고
    • Constitutive activation of STAT3 promotes in-vivo amplification of murine hematopoietic stem cell
    • Abstract 325
    • Oh I-H, Chung Y-J, Eaves CJ, Park B-B, Kang Y-J. Constitutive activation of STAT3 promotes in-vivo amplification of murine hematopoietic stem cell [abstract]. Blood. 2003;102(suppl 1): 95a. Abstract 325.
    • (2003) Blood , vol.102 , Issue.1 SUPPL.
    • Oh, I.-H.1    Chung, Y.-J.2    Eaves, C.J.3    Park, B.-B.4    Kang, Y.-J.5
  • 59
    • 0034892945 scopus 로고    scopus 로고
    • HOXB4 overexpression mediates very rapid stem cell regeneration and competitive hematopoietic repopulation
    • Antonchuk J, Sauvageau G, Humphries RK. HOXB4 overexpression mediates very rapid stem cell regeneration and competitive hematopoietic repopulation. Exp Hematol. 2001;29:1125-1134.
    • (2001) Exp Hematol , vol.29 , pp. 1125-1134
    • Antonchuk, J.1    Sauvageau, G.2    Humphries, R.K.3
  • 60
    • 0037023528 scopus 로고    scopus 로고
    • HOXB4-induced expansion of adult hematopoietic stem cells ex vivo
    • Antonchuk J, Sauvageau G, Humphries RK. HOXB4-induced expansion of adult hematopoietic stem cells ex vivo. Cell. 2002;109:39-45.
    • (2002) Cell , vol.109 , pp. 39-45
    • Antonchuk, J.1    Sauvageau, G.2    Humphries, R.K.3
  • 61
    • 0036682496 scopus 로고    scopus 로고
    • Deregulated expression of HOXB4 enhances the primitive growth activity of human hematopoietic cells
    • Buske C, Feuring-Buske M, Abramovich C, et al. Deregulated expression of HOXB4 enhances the primitive growth activity of human hematopoietic cells. Blood. 2002;100:862-868.
    • (2002) Blood , vol.100 , pp. 862-868
    • Buske, C.1    Feuring-Buske, M.2    Abramovich, C.3
  • 62
    • 0037370740 scopus 로고    scopus 로고
    • High-level ectopic HOXB4 expression confers a profound in vivo competitive growth advantage on human cord blood CD34+ cells, but impairs lymphomyeloid differentiation
    • Schiedlmeier B, Klump H, Will E, 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.
    • (2003) Blood , vol.101 , pp. 1759-1768
    • Schiedlmeier, B.1    Klump, H.2    Will, E.3
  • 63
    • 0033694305 scopus 로고    scopus 로고
    • Pluripotent, cytokine-dependent, hematopoietic stem cells are immortalized by constitutive Notch 1 signaling
    • Varnum-Finney B, Xu L, Brashem-Stein C, et al. Pluripotent, cytokine-dependent, hematopoietic stem cells are immortalized by constitutive Notch 1 signaling. Nat Med. 2000;6:1278-1281.
    • (2000) Nat Med , vol.6 , pp. 1278-1281
    • Varnum-Finney, B.1    Xu, L.2    Brashem-Stein, C.3
  • 64
    • 0037737728 scopus 로고    scopus 로고
    • A role for Wnt signalling in self-renewal of haematopoietic stem cells
    • Reya T, Duncan AW, Ailles L, et al. A role for Wnt signalling in self-renewal of haematopoietic stem cells. Nature. 2003;423:409-414.
    • (2003) Nature , vol.423 , pp. 409-414
    • Reya, T.1    Duncan, A.W.2    Ailles, L.3
  • 65
    • 0036594445 scopus 로고    scopus 로고
    • Gene therapy to protect haematopoietic cells from cytotoxic cancer drugs
    • Sorrentino BP. Gene therapy to protect haematopoietic cells from cytotoxic cancer drugs (Review). Nat Rev Cancer. 2002;2:431-441.
    • (2002) Nat Rev Cancer , vol.2 , pp. 431-441
    • Sorrentino, B.P.1
  • 66
    • 33344456156 scopus 로고    scopus 로고
    • Pharmacologic control of HOXB4-mediated expansion of murine hematopoietic stem cells: Evidence for a restricted time interval for expansion
    • Abstract 1689
    • Shou Y, Stepanova L, Sorrentino B. Pharmacologic control of HOXB4-mediated expansion of murine hematopoietic stem cells: evidence for a restricted time interval for expansion [abstract]. Blood. 2004;104(part 1):470a. Abstract 1689.
    • (2004) Blood , vol.104 , Issue.1 PART
    • Shou, Y.1    Stepanova, L.2    Sorrentino, B.3
  • 67
    • 0343628721 scopus 로고    scopus 로고
    • Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human betaglobin
    • May C, Rivella S, Callegari J, et al. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human betaglobin. Nature. 2000;406:82-86.
    • (2000) Nature , vol.406 , pp. 82-86
    • May, C.1    Rivella, S.2    Callegari, J.3
  • 68
    • 0035861452 scopus 로고    scopus 로고
    • Correction of sickle cell disease in transgenic mouse models by gene therapy
    • Pawliuk R, Westerman KA, Fabry ME, et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science. 2001;294:2368-2371.
    • (2001) Science , vol.294 , pp. 2368-2371
    • Pawliuk, R.1    Westerman, K.A.2    Fabry, M.E.3
  • 69
    • 0034985885 scopus 로고    scopus 로고
    • Generation of dual resistance to 4-hydroperoxycyclophosphamide and methotrexate by retroviral transfer of the human aldehyde dehydrogenase class 1 gene and a mutated dihydrofolate reductase gene
    • Takebe N, Zhao SC, Adhikari D, et al. Generation of dual resistance to 4-hydroperoxycyclophosphamide and methotrexate by retroviral transfer of the human aldehyde dehydrogenase class 1 gene and a mutated dihydrofolate reductase gene. Mol Ther. 2001;3:88-96.
    • (2001) Mol Ther , vol.3 , pp. 88-96
    • Takebe, N.1    Zhao, S.C.2    Adhikari, D.3
  • 70
    • 0031740014 scopus 로고    scopus 로고
    • Retroviral transfer and long-term expression of human cytidine deaminase cDNA in hematopoietic cells following transplantation in mice
    • Eliopoulos N, Bovenzi V, Le NL, et al. Retroviral transfer and long-term expression of human cytidine deaminase cDNA in hematopoietic cells following transplantation in mice. Gene Ther. 1998;5:1545-1551.
    • (1998) Gene Ther , vol.5 , pp. 1545-1551
    • Eliopoulos, N.1    Bovenzi, V.2    Le, N.L.3
  • 71
    • 0029800571 scopus 로고    scopus 로고
    • The hematological effects of folate analogs: Implications for using the dihydrofolate reductase gene for in vivo selection
    • Blau CA, Neff T, Papayannopoulou T. The hematological effects of folate analogs: implications for using the dihydrofolate reductase gene for in vivo selection. Hum Gene Ther. 1996;7:2069-2078.
    • (1996) Hum Gene Ther , vol.7 , pp. 2069-2078
    • Blau, C.A.1    Neff, T.2    Papayannopoulou, T.3
  • 72
    • 0030783511 scopus 로고    scopus 로고
    • Sensitization of hematopoietic stem and progenitor cells to trimetrexate using nucleoside transport inhibitors
    • Allay JA, Spencer HT, Wilkinson SL, Belt JA, Blakley RL, Sorrentino BP. Sensitization of hematopoietic stem and progenitor cells to trimetrexate using nucleoside transport inhibitors. Blood. 1997;90:3546-3554.
    • (1997) Blood , vol.90 , pp. 3546-3554
    • Allay, J.A.1    Spencer, H.T.2    Wilkinson, S.L.3    Belt, J.A.4    Blakley, R.L.5    Sorrentino, B.P.6
  • 73
    • 0031721636 scopus 로고    scopus 로고
    • In vivo selection of retrovirally transduced hematopoietic stem cells
    • Allay JA, Persons DA, Galipeau J, et al. In vivo selection of retrovirally transduced hematopoietic stem cells. Nat Med. 1998;4:1136-1143.
    • (1998) Nat Med , vol.4 , pp. 1136-1143
    • Allay, J.A.1    Persons, D.A.2    Galipeau, J.3
  • 74
    • 0026705269 scopus 로고
    • Transfer and expression of the human multiple drug resistance gene into live mice
    • U S A
    • Podda S, Ward M, Himelstein A, et al. Transfer and expression of the human multiple drug resistance gene into live mice. Proc Natl Acad Sci U S A. 1992;89:9676-9680.
    • (1992) Proc Natl Acad Sci , vol.89 , pp. 9676-9680
    • Podda, S.1    Ward, M.2    Himelstein, A.3
  • 75
    • 0026637623 scopus 로고
    • Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1
    • Sorrentino BP, Brandt SJ, Bodine D, et al. Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1. Science. 1992;257:99-103.
    • (1992) Science , vol.257 , pp. 99-103
    • Sorrentino, B.P.1    Brandt, S.J.2    Bodine, D.3
  • 76
    • 0036169083 scopus 로고    scopus 로고
    • Multidrug resistance 1 gene transfer can confer chemoprotection to human peripheral blood progenitor cells engrafted in immunodeficient mice
    • Schiedlmeier B, Schilz AJ, Kuhlcke K, et al. Multidrug resistance 1 gene transfer can confer chemoprotection to human peripheral blood progenitor cells engrafted in immunodeficient mice. Hum Gene Ther. 2002;13:233-242.
    • (2002) Hum Gene Ther , vol.13 , pp. 233-242
    • Schiedlmeier, B.1    Schilz, A.J.2    Kuhlcke, K.3
  • 77
    • 0032809319 scopus 로고    scopus 로고
    • Paclitaxel chemotherapy after autologous stem-cell transplantation and engraftment of hematopoietic cells transduced with a retrovirus containing the multidrug resistance complementary DNA (MDR1) in metastatic breast cancer patients
    • Cowan KH, Moscow JA, Huang H, et al. Paclitaxel chemotherapy after autologous stem-cell transplantation and engraftment of hematopoietic cells transduced with a retrovirus containing the multidrug resistance complementary DNA (MDR1) in metastatic breast cancer patients. Clin Cancer Res. 1999;5:1619-1628.
    • (1999) Clin Cancer Res , vol.5 , pp. 1619-1628
    • Cowan, K.H.1    Moscow, J.A.2    Huang, H.3
  • 78
    • 0031972859 scopus 로고    scopus 로고
    • Phase I trial of retroviral-mediated transfer of the human MDR1 gene as marrow chemoprotection in patients undergoing high-dose chemotherapy and autologous stem-cell transplantation
    • Hesdorffer C, Ayello J, Ward M, et al. Phase I trial of retroviral-mediated transfer of the human MDR1 gene as marrow chemoprotection in patients undergoing high-dose chemotherapy and autologous stem-cell transplantation. J Clin Oncol. 1998;16:165-172.
    • (1998) J Clin Oncol , vol.16 , pp. 165-172
    • Hesdorffer, C.1    Ayello, J.2    Ward, M.3
  • 79
    • 0030448053 scopus 로고    scopus 로고
    • Results of MDR-1 vector modification trial indicate that granulocyte/macrophage colony-forming unit cells do not contribute to posttransplant hematopoietic recovery following intensive systemic therapy
    • U S A
    • Hanania EG, Giles RE, Kavanagh J, et al. Results of MDR-1 vector modification trial indicate that granulocyte/macrophage colony-forming unit cells do not contribute to posttransplant hematopoietic recovery following intensive systemic therapy. Proc Natl Acad Sci U S A. 1996;93:15346-15351.
    • (1996) Proc Natl Acad Sci , vol.93 , pp. 15346-15351
    • Hanania, E.G.1    Giles, R.E.2    Kavanagh, J.3
  • 80
    • 0031020809 scopus 로고    scopus 로고
    • Cytokine prestimulation as a gene therapy strategy: Implications for using the MDR1 gene as a dominant selectable marker
    • Blau CA, Neff T, Papayannopoulou T. Cytokine prestimulation as a gene therapy strategy: implications for using the MDR1 gene as a dominant selectable marker. Blood. 1997;89:146-154.
    • (1997) Blood , vol.89 , pp. 146-154
    • Blau, C.A.1    Neff, T.2    Papayannopoulou, T.3
  • 81
    • 0032188998 scopus 로고    scopus 로고
    • 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
    • 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.
    • (1998) Blood , vol.92 , pp. 2269-2279
    • Bunting, K.D.1    Galipeau, J.2    Topham, D.3    Benaim, E.4    Sorrentino, B.P.5
  • 82
    • 2942700669 scopus 로고    scopus 로고
    • Reproducible insertional leukemogenesis with evidence for chromosomal instability following dose-escalated retroviral vector transfer into murine hematopoietic cells
    • Abstract 699
    • Modlich U, Meyer J, Rudolph C, et al. Reproducible insertional leukemogenesis with evidence for chromosomal instability following dose-escalated retroviral vector transfer into murine hematopoietic cells [abstract]. Blood. 2003;102(part 1):200a. Abstract 699.
    • (2003) Blood , vol.102 , Issue.1 PART
    • Modlich, U.1    Meyer, J.2    Rudolph, C.3
  • 83
    • 21144437399 scopus 로고    scopus 로고
    • Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis
    • Modlich U, Kustikova OS, Schmidt M, et al. Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis. Blood. 2005;105:4235-4246.
    • (2005) Blood , vol.105 , pp. 4235-4246
    • Modlich, U.1    Kustikova, O.S.2    Schmidt, M.3
  • 84
    • 0028794564 scopus 로고
    • Structure, function, and inhibition of O6-alkylguanine-DNA alkyltransferase
    • Pegg AE, Dolan ME, Moschel RC. Structure, function, and inhibition of O6-alkylguanine-DNA alkyltransferase (Review). Prog Nucl Acid Res Mol Biol. 1995;51:167-223.
    • (1995) Prog Nucl Acid Res Mol Biol , vol.51 , pp. 167-223
    • Pegg, A.E.1    Dolan, M.E.2    Moschel, R.C.3
  • 85
    • 0031595389 scopus 로고    scopus 로고
    • Correlation of tumor O6 methylguanine-DNA methyltransferase levels with survival of malignant astrocytoma patients treated with bis- chloroethylnitrosourea: A Southwest Oncology Group study
    • Jaeckle KA, Eyre HJ, Townsend JJ, et al. Correlation of tumor O6 methylguanine-DNA methyltransferase levels with survival of malignant astrocytoma patients treated with bis-chloroethylnitrosourea: a Southwest Oncology Group study. J Clin Oncol. 1998;16:3310-3315.
    • (1998) J Clin Oncol , vol.16 , pp. 3310-3315
    • Jaeckle, K.A.1    Eyre, H.J.2    Townsend, J.J.3
  • 86
    • 0034626988 scopus 로고    scopus 로고
    • Inactivation of the DNA-repair gene MGMT and the clinical response of gliomas to alkylating agents
    • erratum appears in N Engl J Med. 2000;343:1740
    • Esteller M, Garcia-Foncillas J, Andion E, et al. Inactivation of the DNA-repair gene MGMT and the clinical response of gliomas to alkylating agents [erratum appears in N Engl J Med. 2000;343:1740]. N Engl J Med. 2000;343:1350-1354.
    • (2000) N Engl J Med , vol.343 , pp. 1350-1354
    • Esteller, M.1    Garcia-Foncillas, J.2    Andion, E.3
  • 87
    • 12144291692 scopus 로고    scopus 로고
    • Clinical trial substantiates the predictive value of O-6-methylguanine- DNA methyltransferase promoter methylation in glioblastoma patients treated with temozolomide
    • Hegi ME, Diserens AC, Godard S, et al. Clinical trial substantiates the predictive value of O-6-methylguanine-DNA methyltransferase promoter methylation in glioblastoma patients treated with temozolomide. Clin Cancer Res. 2004;10:1871-1874.
    • (2004) Clin Cancer Res , vol.10 , pp. 1871-1874
    • Hegi, M.E.1    Diserens, A.C.2    Godard, S.3
  • 88
    • 0029056481 scopus 로고
    • Retroviral transduction and expression of the human alkyltransferase cDNA provides nitrosourea resistance to hematopoietic cells
    • Allay JA, Dumenco LL, Koc ON, Liu L, Gerson SL. Retroviral transduction and expression of the human alkyltransferase cDNA provides nitrosourea resistance to hematopoietic cells. Blood. 1995;85:3342-3351.
    • (1995) Blood , vol.85 , pp. 3342-3351
    • Allay, J.A.1    Dumenco, L.L.2    Koc, O.N.3    Liu, L.4    Gerson, S.L.5
  • 89
    • 0030445950 scopus 로고    scopus 로고
    • Retroviral transduction of a mutant methylguanine DNA methyltransferase gene into human CD34 cells confers resistance to O6-benzylguanine plus 1,3-bis(2-chloroethyl)-1-nitrosourea
    • U S A
    • Reese JS, Koc ON, Lee KM, et al. Retroviral transduction of a mutant methylguanine DNA methyltransferase gene into human CD34 cells confers resistance to O6-benzylguanine plus 1,3-bis(2-chloroethyl)-1-nitrosourea. Proc Natl Acad Sci U S A. 1996;93:14088-14093.
    • (1996) Proc Natl Acad Sci , vol.93 , pp. 14088-14093
    • Reese, J.S.1    Koc, O.N.2    Lee, K.M.3
  • 90
    • 0025924488 scopus 로고
    • Effect of O6-benzylguanine analogues on sensitivity of human tumor cells to the cytotoxic effects of alkylating agents
    • Dolan ME, Mitchell RB, Mummert C, Moschel RC, Pegg AE. Effect of O6-benzylguanine analogues on sensitivity of human tumor cells to the cytotoxic effects of alkylating agents. Cancer Res. 1991;51:3367-3372.
    • (1991) Cancer Res , vol.51 , pp. 3367-3372
    • Dolan, M.E.1    Mitchell, R.B.2    Mummert, C.3    Moschel, R.C.4    Pegg, A.E.5
  • 91
    • 0028979534 scopus 로고
    • Mutations in the Ada O6-alkylguanine-DNA alkyltransferase conferring sensitivity to inactivation by O6-benzylguanine and 2,4-diamino-6-benzyloxy-5- nitrosopyrimidine
    • Crone TM, Kanugula S, Pegg AE. Mutations in the Ada O6-alkylguanine-DNA alkyltransferase conferring sensitivity to inactivation by O6-benzylguanine and 2,4-diamino-6-benzyloxy-5-nitrosopyrimidine. Carcinogenesis. 1995;16:1687-1692.
    • (1995) Carcinogenesis , vol.16 , pp. 1687-1692
    • Crone, T.M.1    Kanugula, S.2    Pegg, A.E.3
  • 92
    • 0033566313 scopus 로고    scopus 로고
    • Protection of CHO cells by mutant forms of O6-alkylguanine-DNA alkyltransferase from killing by 1,3-bis-(2-chloroethyl)-1-nitrosourea (BCNU) plus O6-benzylguanine or O6-benzyl-8-oxoguanine
    • Loktionova NA, Xu-Welliver M, Crone TM, Kanugula S, Pegg AE. Protection of CHO cells by mutant forms of O6-alkylguanine-DNA alkyltransferase from killing by 1,3-bis-(2-chloroethyl)-1-nitrosourea (BCNU) plus O6-benzylguanine or O6-benzyl-8-oxoguanine. Biochem Pharmacol. 1999;58:237-244.
    • (1999) Biochem Pharmacol , vol.58 , pp. 237-244
    • Loktionova, N.A.1    Xu-Welliver, M.2    Crone, T.M.3    Kanugula, S.4    Pegg, A.E.5
  • 93
    • 0034657950 scopus 로고    scopus 로고
    • Limiting numbers of G156A O(6)-methylguanine-DNA methyltransferase- transduced marrow progenitors repopulate nonmyeloablated mice after drug selection
    • 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.
    • (2000) Blood , vol.95 , pp. 3078-3084
    • Davis, B.M.1    Koc, O.N.2    Gerson, S.L.3
  • 94
    • 0034665336 scopus 로고    scopus 로고
    • Direct reversal of DNA damage by mutant methyltransferase protein protects mice against dose-intensi-fied chemotherapy and leads to in vivo selection of hematopoietic stem cells
    • Ragg S, Xu-Welliver M, Bailey J, et al. Direct reversal of DNA damage by mutant methyltransferase protein protects mice against dose-intensi-fied chemotherapy and leads to in vivo selection of hematopoietic stem cells. Cancer Res. 2000;60:5187-5195.
    • (2000) Cancer Res , vol.60 , pp. 5187-5195
    • Ragg, S.1    Xu-Welliver, M.2    Bailey, J.3
  • 95
    • 10744232285 scopus 로고    scopus 로고
    • In vivo selection of human hematopoietic cells in a xenograft model using combined pharmacologic and genetic manipulations
    • Pollok KE, Hartwell JR, Braber A, et al. In vivo selection of human hematopoietic cells in a xenograft model using combined pharmacologic and genetic manipulations. Hum Gene Ther. 2003;14:1703-1714.
    • (2003) Hum Gene Ther , vol.14 , pp. 1703-1714
    • Pollok, K.E.1    Hartwell, J.R.2    Braber, A.3
  • 96
    • 0347086143 scopus 로고    scopus 로고
    • In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning
    • 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.
    • (2003) J Clin Invest , vol.112 , pp. 1561-1570
    • Zielske, S.P.1    Reese, J.S.2    Lingas, K.T.3    Donze, J.R.4    Gerson, S.L.5
  • 97
    • 0034672163 scopus 로고    scopus 로고
    • Similar pattern of thymic-dependent T-cell reconstitution in infants with severe combined immunodeficiency after human leukocyte antigen (HLA)-identical and HLA-nonidentical stem cell transplantation
    • Muller SM, Kohn T, Schulz AS, Debatin KM, Friedrich W. Similar pattern of thymic-dependent T-cell reconstitution in infants with severe combined immunodeficiency after human leukocyte antigen (HLA)-identical and HLA-nonidentical stem cell transplantation. Blood. 2000;96:4344-4349.
    • (2000) Blood , vol.96 , pp. 4344-4349
    • Muller, S.M.1    Kohn, T.2    Schulz, A.S.3    Debatin, K.M.4    Friedrich, W.5
  • 98
    • 18244411938 scopus 로고    scopus 로고
    • Longterm immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: A European retrospective study of 116 patients
    • Haddad E, Landais P, Friedrich W, et al. Longterm immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: a European retrospective study of 116 patients. Blood. 1998;91:3646-3653.
    • (1998) Blood , vol.91 , pp. 3646-3653
    • Haddad, E.1    Landais, P.2    Friedrich, W.3
  • 99
    • 0029902033 scopus 로고    scopus 로고
    • Spontaneous in vivo reversion to normal of an inherited mutation in a patient with adenosine deaminase deficiency
    • Hirschhorn R, Yang DR, Puck JM, Huie ML, Jiang CK, Kurlandsky LE. Spontaneous in vivo reversion to normal of an inherited mutation in a patient with adenosine deaminase deficiency. Nat Genet. 1996;13:290-295.
    • (1996) Nat Genet , vol.13 , pp. 290-295
    • Hirschhorn, R.1    Yang, D.R.2    Puck, J.M.3    Huie, M.L.4    Jiang, C.K.5    Kurlandsky, L.E.6
  • 100
    • 12944284582 scopus 로고    scopus 로고
    • Diversity, functionality, and stability of the T cell repertoire derived in vivo from a single human T cell precursor
    • U S A
    • Bousso P, Wahn V, Douagi I, et al. Diversity, functionality, and stability of the T cell repertoire derived in vivo from a single human T cell precursor. Proc Natl Acad Sci U S A. 2000;97:274-278.
    • (2000) Proc Natl Acad Sci , vol.97 , pp. 274-278
    • Bousso, P.1    Wahn, V.2    Douagi, I.3
  • 101
    • 0035877972 scopus 로고    scopus 로고
    • Variable correction of host defense following gene transfer and bone marrow transplantation in murine X-linked chronic granulomatous disease
    • Dinauer MC, Gifford MA, Pech N, Li LL, Emshwiller P. Variable correction of host defense following gene transfer and bone marrow transplantation in murine X-linked chronic granulomatous disease. Blood. 2001;97:3738-3745.
    • (2001) Blood , vol.97 , pp. 3738-3745
    • Dinauer, M.C.1    Gifford, M.A.2    Pech, N.3    Li, L.L.4    Emshwiller, P.5
  • 102
    • 33344467079 scopus 로고    scopus 로고
    • Chronic granulomatous disease gene therapy functionally corrects the phenotype of polymorphonuclear leukocytes (PMN)
    • Abstract 332
    • Siler U, Ott MG, Stein S, et al. Chronic granulomatous disease gene therapy functionally corrects the phenotype of polymorphonuclear leukocytes (PMN) [abstract]. Mol Ther. 2005;11(suppl 1):S129. Abstract 332.
    • (2005) Mol Ther , vol.11 , Issue.1 SUPPL.
    • Siler, U.1    Ott, M.G.2    Stein, S.3
  • 103
    • 0036819773 scopus 로고    scopus 로고
    • Update on hematopoietic stem cell gene transfer using non-human primate models
    • Hu J, Dunbar CE. Update on hematopoietic stem cell gene transfer using non-human primate models (Review). Curr Opin Mol Ther. 2002;4:482-490.
    • (2002) Curr Opin Mol Ther , vol.4 , pp. 482-490
    • Hu, J.1    Dunbar, C.E.2
  • 104
    • 4444313651 scopus 로고    scopus 로고
    • Stem cell gene transfer: Efficacy and safety in large animal studies
    • Horn PA, Morris JC, Neff T, Kiem H-P. Stem cell gene transfer: efficacy and safety in large animal studies. Mol Ther. 2004;10:417-431.
    • (2004) Mol Ther , vol.10 , pp. 417-431
    • Horn, P.A.1    Morris, J.C.2    Neff, T.3    Kiem, H.-P.4
  • 105
    • 0027263351 scopus 로고
    • Long-term engraftment of normal and post-5-fluorouracil murine marrow in normal nonmyeloablated mice
    • Stewart FM, Crittenden RB, Lowry PA, Pearson-White S, Quesenberry PJ. Long-term engraftment of normal and post-5-fluorouracil murine marrow in normal nonmyeloablated mice. Blood. 1993;81:2566-2571.
    • (1993) Blood , vol.81 , pp. 2566-2571
    • Stewart, F.M.1    Crittenden, R.B.2    Lowry, P.A.3    Pearson-White, S.4    Quesenberry, P.J.5
  • 106
    • 0942266035 scopus 로고    scopus 로고
    • Lentivector-mediated clonal tracking reveals intrinsic heterogeneity in the human hematopoietic stem cell compartment and culture-induced stem cell impairment
    • Mazurier F, Gan OI, McKenzie JL, Doedens M, Dick JE. Lentivector-mediated clonal tracking reveals intrinsic heterogeneity in the human hematopoietic stem cell compartment and culture-induced stem cell impairment. Blood. 2004;103:545-552.
    • (2004) Blood , vol.103 , pp. 545-552
    • Mazurier, F.1    Gan, O.I.2    McKenzie, J.L.3    Doedens, M.4    Dick, J.E.5
  • 108
    • 21144439712 scopus 로고    scopus 로고
    • Failure of SCID-X1 gene therapy in older patients
    • Thrasher AJ, Hacein-Bey-Abina S, Gaspar HB, et al. Failure of SCID-X1 gene therapy in older patients. Blood. 2005;105:4255-4257.
    • (2005) Blood , vol.105 , pp. 4255-4257
    • Thrasher, A.J.1    Hacein-Bey-Abina, S.2    Gaspar, H.B.3
  • 109
    • 0141744749 scopus 로고    scopus 로고
    • Reduction in hematopoietic stem cell numbers with in vivo drug selection can be partially abrogated by HOXB4 gene expression
    • Sawai N, Persons DA, Zhou S, Lu T, Sorrentino BP. Reduction in hematopoietic stem cell numbers with in vivo drug selection can be partially abrogated by HOXB4 gene expression. Mol Ther. 2003;8:376-384.
    • (2003) Mol Ther , vol.8 , pp. 376-384
    • Sawai, N.1    Persons, D.A.2    Zhou, S.3    Lu, T.4    Sorrentino, B.P.5
  • 110
    • 0029796633 scopus 로고    scopus 로고
    • Long-term lymphohematopoietic reconstitution by a single CD34-low/negative hematopoietic stem cell
    • Osawa M, Hanada K, Hamada H, Nakauchi H. Long-term lymphohematopoietic reconstitution by a single CD34-low/negative hematopoietic stem cell. Science. 1996;273:242-245.
    • (1996) Science , vol.273 , pp. 242-245
    • Osawa, M.1    Hanada, K.2    Hamada, H.3    Nakauchi, H.4
  • 111
    • 0028953448 scopus 로고
    • Behavior of hematopoietic stem cells in a large animal
    • U S A
    • Abkowitz JL, Persik MT, Shelton GH, et al. Behavior of hematopoietic stem cells in a large animal. Proc Natl Acad Sci U S A. 1995;92:2031-2035.
    • (1995) Proc Natl Acad Sci , vol.92 , pp. 2031-2035
    • Abkowitz, J.L.1    Persik, M.T.2    Shelton, G.H.3
  • 112
    • 0030065744 scopus 로고    scopus 로고
    • Evidence that hematopoiesis may be a stochastic process in vivo
    • Abkowitz JL, Catlin SN, Guttorp P. Evidence that hematopoiesis may be a stochastic process in vivo. Nat Med. 1996;2:190-197.
    • (1996) Nat Med , vol.2 , pp. 190-197
    • Abkowitz, J.L.1    Catlin, S.N.2    Guttorp, P.3
  • 113
    • 0037370002 scopus 로고    scopus 로고
    • T cell repertoire development in humans with SCID after nonablative allogeneic marrow transplantation
    • Sarzotti M, Patel DD, Li X, et al. T cell repertoire development in humans with SCID after nonablative allogeneic marrow transplantation. J Immunol. 2003;170:2711-2718.
    • (2003) J Immunol , vol.170 , pp. 2711-2718
    • Sarzotti, M.1    Patel, D.D.2    Li, X.3
  • 114
    • 0345144017 scopus 로고    scopus 로고
    • Clonality analysis after retroviral-mediated gene transfer to CD34(+) cells from the cord blood of ADA-deficient SCID neonates
    • Schmidt M, Carbonaro DA, Speckmann C, et al. Clonality analysis after retroviral-mediated gene transfer to CD34(+) cells from the cord blood of ADA-deficient SCID neonates. Nat Med. 2003;9:463-468.
    • (2003) Nat Med , vol.9 , pp. 463-468
    • Schmidt, M.1    Carbonaro, D.A.2    Speckmann, C.3
  • 115
    • 0037134031 scopus 로고    scopus 로고
    • Murine leukemia induced by retroviral gene marking [Brevia]
    • Li Z, Dullmann J, Schiedlmeier B, et al. Murine leukemia induced by retroviral gene marking [Brevia]. Science. 2002;296:497.
    • (2002) Science , vol.296 , pp. 497
    • Li, Z.1    Dullmann, J.2    Schiedlmeier, B.3
  • 116
    • 21044448037 scopus 로고    scopus 로고
    • Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking
    • Kustikova O, Fehse B, Modlich U, et al. Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science. 2005;308:1171-1174.
    • (2005) Science , vol.308 , pp. 1171-1174
    • Kustikova, O.1    Fehse, B.2    Modlich, U.3
  • 117
    • 12144289202 scopus 로고    scopus 로고
    • Longterm clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: No progression to clonal hematopoiesis or leukemia
    • Kiem H-P, Sellers S, Thomasson B, et al. Longterm 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;9:389-395.
    • (2004) Mol Ther , vol.9 , pp. 389-395
    • Kiem, H.-P.1    Sellers, S.2    Thomasson, B.3
  • 118
    • 0347634456 scopus 로고    scopus 로고
    • Gene therapy insertional mutagenesis insights [Brevia]
    • Dave UP, Jenkins NA, Copeland NG. Gene therapy insertional mutagenesis insights [Brevia]. Science. 2004;303:333.
    • (2004) Science , vol.303 , pp. 333
    • Dave, U.P.1    Jenkins, N.A.2    Copeland, N.G.3
  • 119
    • 33344460479 scopus 로고    scopus 로고
    • Mouse models for the analysis of genetic risk factors for hematopoietic transformation in XSCID gene therapy
    • Abstract 454
    • Shou Y, Stepanova L, Sorrentino BP. Mouse models for the analysis of genetic risk factors for hematopoietic transformation in XSCID gene therapy [abstract]. Mol Ther. 2005;11(suppl 1): S176. Abstract 454.
    • (2005) Mol Ther , vol.11 , Issue.1 SUPPL.
    • Shou, Y.1    Stepanova, L.2    Sorrentino, B.P.3
  • 120
    • 0035869537 scopus 로고    scopus 로고
    • Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: Collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program
    • Filipovich AH, Stone JV, Tomany SC, et al. Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program. Blood. 2001;97:1598-1603.
    • (2001) Blood , vol.97 , pp. 1598-1603
    • Filipovich, A.H.1    Stone, J.V.2    Tomany, S.C.3
  • 121
    • 0034028630 scopus 로고    scopus 로고
    • Unrelated bone marrow transplantation in children: Outcome and a comparison with sibling donor grafting
    • Gustafsson A, Remberger M, Winiarski J, Ringden O. Unrelated bone marrow transplantation in children: outcome and a comparison with sibling donor grafting. Bone Marrow Transplant. 2000;25:1059-1065.
    • (2000) Bone Marrow Transplant , vol.25 , pp. 1059-1065
    • Gustafsson, A.1    Remberger, M.2    Winiarski, J.3    Ringden, O.4


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