메뉴 건너뛰기




Volumn 102, Issue 5, 2009, Pages 357-363

The evolution of gene therapy in X-linked severe combined immunodeficiency

Author keywords

[No Author keywords available]

Indexed keywords

ANTIBIOTIC AGENT; CYTOKINE RECEPTOR GAMMA CHAIN; IMMUNOGLOBULIN; RECEPTOR SUBUNIT; RETROVIRUS VECTOR; UNCLASSIFIED DRUG;

EID: 66249113524     PISSN: 10811206     EISSN: 15344436     Source Type: Journal    
DOI: 10.1016/S1081-1206(10)60504-2     Document Type: Review
Times cited : (9)

References (54)
  • 1
    • 0027403374 scopus 로고
    • Interleukin-2 receptor-chain mutation results in X-linked severe combined immunodeficiency in humans
    • Noguchi M, Yi H, Rosenblart HM, et al. Interleukin-2 receptor-chain mutation results in X-linked severe combined immunodeficiency in humans Cell. 1993;73:147-157.
    • (1993) Cell , vol.73 , pp. 147-157
    • Noguchi, M.1    Yi, H.2    Rosenblart, H.M.3
  • 2
    • 0027320217 scopus 로고
    • The interleukin-2 receptor-chain maps to Xq13.1 and is mutated in X-linked severe combined immunodeficiency, XSCID1
    • Puck JM, Deschenes SM, Porter JC, et al. The interleukin-2 receptor-chain maps to Xq13.1 and is mutated in X-linked severe combined immunodeficiency, XSCID1. Hum Mol Genet. 1992;2:1099 -1104.
    • (1992) Hum Mol Genet , vol.2 , pp. 1099-1104
    • Puck, J.M.1    Deschenes, S.M.2    Porter, J.C.3
  • 3
    • 0028978898 scopus 로고
    • The common--chain for multiple cytokine receptors
    • Sugamura K, Asao H, Kondo M, et al. The common--chain for multiple cytokine receptors Adv Immunol. 1995;59:225-277.
    • (1995) Adv Immunol , vol.59 , pp. 225-277
    • Sugamura, K.1    Asao, H.2    Kondo, M.3
  • 5
    • 0029414787 scopus 로고
    • Common cytokine receptor gamma chain (gamma c)-dependent cytokines: Understanding in vivo functions by gene targeting
    • Di Santo JP, Kuhn R, Muller W. Common cytokine receptor gamma chain (gamma c)-dependent cytokines: understanding in vivo functions by gene targeting. Immunol Rev. 1995;148:19 -34.
    • (1995) Immunol Rev , vol.148 , pp. 19-34
    • Di Santo, J.P.1    Kuhn, R.2    Muller, W.3
  • 7
    • 0014345361 scopus 로고
    • Phenotypic expressions of the major histocompatibility locus in man (HL-A): Leukocyte antigens and mixed leukocyte culture reactivity
    • Amos DB, Bach FH. Phenotypic expressions of the major histocompatibility locus in man (HL-A): leukocyte antigens and mixed leukocyte culture reactivity, J Exp Med. 1968;128:623- 637.
    • (1968) J Exp Med , vol.128 , pp. 623-637
    • Amos, D.B.1    Bach, F.H.2
  • 8
    • 0039248549 scopus 로고    scopus 로고
    • T cell depleted haploidentical bone marrow transplantation for the treatment of children with severe combined immunodeficiency
    • Smogorzewska EM, Brooks J, Annett G, et al. T cell depleted haploidentical bone marrow transplantation for the treatment of children with severe combined immunodeficiency. Arch Immunol Ther Exp (Warsz). 2000;48:111-118.
    • (2000) Arch Immunol Ther Exp (Warsz) , vol.48 , pp. 111-118
    • Smogorzewska, E.M.1    Brooks, J.2    Annett, G.3
  • 9
    • 0037442176 scopus 로고    scopus 로고
    • Long-term survival and transplantation of hematopoietic stem cells for immunodeficiencies: Report of the European experience 1968-1999l
    • Antoine C, Suller S, Cant A, et al. Long-term survival and transplantation of hematopoietic stem cells for immunodeficiencies: report of the European experience 1968-1999l. Lancet. 2003;361:553-556.
    • (2003) Lancet , vol.361 , pp. 553-556
    • Antoine, C.1    Suller, S.2    Cant, A.3
  • 10
    • 0038268108 scopus 로고    scopus 로고
    • Risks of mortality in children admitted to the paediatric intensive care unit after haematopoietic stem cell transplantation
    • Tomaske M, Bosk A, Eyrich M, et al. Risks of mortality in children admitted to the paediatric intensive care unit after haematopoietic stem cell transplantation. Br J Haematol. 2003;121:886-891.
    • (2003) Br J Haematol , vol.121 , pp. 886-891
    • Tomaske, M.1    Bosk, A.2    Eyrich, M.3
  • 11
    • 0034446692 scopus 로고    scopus 로고
    • Advances in the understanding and treatment of human severe combined immunodeficiency
    • Buckley RH. Advances in the understanding and treatment of human severe combined immunodeficiency, Immunol Res. 2001;22:237-251.
    • (2001) Immunol Res , vol.22 , pp. 237-251
    • Buckley, R.H.1
  • 12
    • 0014433553 scopus 로고
    • Immunological reconstitution of sex-linked lymphopenic immunological deficiency
    • Gatti RA, Meuwissen HJ, Allen HD, et al. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet. 1968;2: 1366-1369.
    • (1968) Lancet , vol.2 , pp. 1366-1369
    • Gatti, R.A.1    Meuwissen, H.J.2    Allen, H.D.3
  • 13
    • 18244411938 scopus 로고    scopus 로고
    • Long-term 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. Long-term 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
  • 14
    • 0033580206 scopus 로고    scopus 로고
    • Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency
    • Buckley RH, Schiff SE, Schiff, RI, et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med. 1999;340:508 -516.
    • (1999) N Engl J Med , vol.340 , pp. 508-516
    • Buckley, R.H.1    Schiff, S.E.2    Schiff, R.I.3
  • 16
    • 0036384319 scopus 로고    scopus 로고
    • Gene therapy in infants with severe combined immunodeficiency
    • Otsu M, Candotti F. Gene therapy in infants with severe combined immunodeficiency. Biodrugs. 2002;16:229 -239.
    • (2002) Biodrugs , vol.16 , pp. 229-239
    • Otsu, M.1    Candotti, F.2
  • 17
    • 0033512908 scopus 로고    scopus 로고
    • Influence of severe combined immunodeficiency phenotype on the outcome of HAL non-identical, T-cell-depleted bone marrow transplantation: A retrospective European survey from the European Group for Bone Marrow Transplantation and the European Society for Immunodeficiency
    • Bertrand Y, Ladais P, Friedrich W, et al. Influence of severe combined immunodeficiency phenotype on the outcome of HAL non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European Group for Bone Marrow Transplantation and the European Society for Immunodeficiency J Pediatr. 1999;134:740-748.
    • (1999) J Pediatr , vol.134 , pp. 740-748
    • Bertrand, Y.1    Ladais, P.2    Friedrich, W.3
  • 18
    • 0032533265 scopus 로고    scopus 로고
    • Engraftment of severe combined immune deficient mice receiving allogeneic bone marrow via in utero postnatal transfer
    • Blazar BR, Taylor PA, McElmurry R, et al. Engraftment of severe combined immune deficient mice receiving allogeneic bone marrow via in utero postnatal transfer. Blood. 1998;92:3949-3959.
    • (1998) Blood , vol.92 , pp. 3949-3959
    • Blazar, B.R.1    Taylor, P.A.2    McElmurry, R.3
  • 19
    • 1942435994 scopus 로고    scopus 로고
    • Successes and risks of gene therapy in primary immunodeficiencies
    • Chinen J, Puck JM. Successes and risks of gene therapy in primary immunodeficiencies. J Allergy Clin Immunol. 2004;113:595- 603.
    • (2004) J Allergy Clin Immunol , vol.113 , pp. 595-603
    • Chinen, J.1    Puck, J.M.2
  • 20
    • 0037424875 scopus 로고    scopus 로고
    • Current developments in the design of oncoretrovirus and lentivirus vector systems for hematopoietic cell gene therapy
    • Brenner S, Malech HL. Current developments in the design of oncoretrovirus and lentivirus vector systems for hematopoietic cell gene therapy. Biochim Biophys Acta. 2003;1640:1-24.
    • (2003) Biochim Biophys Acta , vol.1640 , pp. 1-24
    • Brenner, S.1    Malech, H.L.2
  • 21
    • 0034604048 scopus 로고    scopus 로고
    • Thymic function after hematopoieticstem-cell transplantation for the treatment of severe combined immunodeficiency
    • Patel DD, Gooding ME, Parrott RE, et al. Thymic function after hematopoieticstem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med. 2000;342:1325-1332.
    • (2000) N Engl J Med , vol.342 , pp. 1325-1332
    • Patel, D.D.1    Gooding, M.E.2    Parrott, R.E.3
  • 24
    • 0242286486 scopus 로고    scopus 로고
    • Primer on medical genomics, part X: Genetherapy
    • Russell SJ, Peng KW. Primer on medical genomics, part X: genetherapy. Mayo Clin Proc. 2003;78:1370-1383.
    • (2003) Mayo Clin Proc , vol.78 , pp. 1370-1383
    • Russell, S.J.1    Peng, K.W.2
  • 25
    • 0030788390 scopus 로고    scopus 로고
    • Gene transfer to hematopoietic cells: The clinical experience
    • Richter J. Gene transfer to hematopoietic cells: the clinical experience. Eur J Haematol. 1997;59:67-75.
    • (1997) Eur J Haematol , vol.59 , pp. 67-75
    • Richter, J.1
  • 26
    • 0033789754 scopus 로고    scopus 로고
    • Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells
    • Sadelain M, Frassoni F, Riviere I. Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells. Curr OpinHematol. 2000;7:364 -377.
    • (2000) Curr OpinHematol , vol.7 , pp. 364-377
    • Sadelain, M.1    Frassoni, F.2    Riviere, I.3
  • 27
    • 0034724857 scopus 로고    scopus 로고
    • Genetherapy of human severe combined immunodeficiency (SCID)-X1 disease
    • Cavazzana-Calvo M, Hacein-Bey S, de Saint Baseile G, et al. Genetherapy 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 Baseile, G.3
  • 28
    • 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
  • 29
    • 19244362468 scopus 로고    scopus 로고
    • Retroviral gene transfer in autologous bone marrow transplantation for adult acute leukemia
    • Cornetta K, Srour EF, Moore A, et al. Retroviral gene transfer in autologous bone marrow transplantation for adult acute leukemia. HumGene Ther. 1996;6:1323-1329.
    • (1996) HumGene Ther , vol.6 , pp. 1323-1329
    • Cornetta, K.1    Srour, E.F.2    Moore, A.3
  • 30
    • 22744455256 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: Development of improved lentiviral and retroviral vectors: design, biosafety, and production
    • Sinn PL, Sauter SL, McCray PB Jr. Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors: design, biosafety, and production. Gene Ther. 2005;12:1089 -1098.
    • (2005) Gene Ther , vol.12 , pp. 1089-1098
    • Sinn, P.L.1    Sauter, S.L.2    McCray Jr., P.B.3
  • 31
    • 0035835375 scopus 로고    scopus 로고
    • Optimization of retroviral gene transfer protocol to maintain the lymphoid potential of progenitor cells
    • Hacein-Bey S, Gross F, Nusbaum P, et al. Optimization of retroviral gene transfer protocol to maintain the lymphoid potential of progenitor cells. Hum Gene Ther. 2001;12:291-301.
    • (2001) Hum Gene Ther , vol.12 , pp. 291-301
    • Hacein-Bey, S.1    Gross, F.2    Nusbaum, P.3
  • 32
    • 16044366110 scopus 로고    scopus 로고
    • Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells
    • Hanenberg H, Xiao XL, Dilloo D, et al. Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells. Nat Med. 1996;2:876-882.
    • (1996) Nat Med , vol.2 , pp. 876-882
    • Hanenberg, H.1    Xiao, X.L.2    Dilloo, D.3
  • 33
    • 0031564330 scopus 로고    scopus 로고
    • Optimization of fibronectinassisted retroviral gene transfer into human CD34-hematopoietic cells
    • Hanenberg H, Hashino K, Konishi H, et al. Optimization of fibronectinassisted retroviral gene transfer into human CD34-hematopoietic cells. Hum Gene Ther. 1997;8:2193-2206.
    • (1997) Hum Gene Ther , vol.8 , pp. 2193-2206
    • Hanenberg, H.1    Hashino, K.2    Konishi, H.3
  • 34
    • 0034501064 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy: Towards clinically significant gene transfer efficiency
    • Heim DA, Dunbar CE. Hematopoietic stem cell gene therapy: towards clinically significant gene transfer efficiency. Immunl Rev. 2000;178:29-38.
    • (2000) Immunl Rev , vol.178 , pp. 29-38
    • Heim, D.A.1    Dunbar, C.E.2
  • 35
    • 10744233858 scopus 로고    scopus 로고
    • American Society of Gene Therapy (ASGT). American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells
    • Kohn DB, Sadelain M, Dunbar C, et al; American Society of Gene Therapy (ASGT). American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther. 2003;8:180 -187.
    • (2003) Mol Ther , vol.8 , pp. 180-187
    • Kohn, D.B.1    Sadelain, M.2    Dunbar, C.3
  • 36
    • 0026641562 scopus 로고
    • Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer
    • Donahue RE, Kessler WE, Bodine D, et al. Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer. J Exp Med. 1992;176:1125-1135.
    • (1992) J Exp Med , vol.176 , pp. 1125-1135
    • Donahue, R.E.1    Kessler, W.E.2    Bodine, D.3
  • 37
    • 0029850719 scopus 로고    scopus 로고
    • Role of interleukin-2 (IL-2), IL-7, IL-15 in natural killer cell differentiation from cord blood hematopoietic progenitor cells and from gamma c tranduced severe combined immunodeficiency X1 bone marrow cells
    • Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Role of interleukin-2 (IL-2), IL-7, IL-15 in natural killer cell differentiation from cord blood hematopoietic progenitor cells and from gamma c tranduced severe combined immunodeficiency X1 bone marrow cells. Blood. 1996;88:3901-3909.
    • (1996) Blood , vol.88 , pp. 3901-3909
    • Cavazzana-Calvo, M.1    Hacein-Bey, S.2    de Saint Basile, G.3
  • 38
    • 0032237804 scopus 로고    scopus 로고
    • γc gene transfer in the presence of stem cell factor, FLT-3L, interleukin-7 (IL-7), IL-1, and IL-15 cytokines restores T-cell differentiation from-c (-) X-linked severe combined immunodeficiency hematopoietic progenitor cells in murine fetal thymic organ cultures
    • Hacein-Bey S, Basile GD, Lemerle J, et al. γc gene transfer in the presence of stem cell factor, FLT-3L, interleukin-7 (IL-7), IL-1, and IL-15 cytokines restores T-cell differentiation from-c (-) X-linked severe combined immunodeficiency hematopoietic progenitor cells in murine fetal thymic organ cultures. Blood. 1998;92:4090-4097.
    • (1998) Blood , vol.92 , pp. 4090-4097
    • Hacein-Bey, S.1    Basile, G.D.2    Lemerle, J.3
  • 39
    • 0028878330 scopus 로고
    • Lymphoid development in mice with a targeted deletion of the interleukin 2 receptor-chain
    • DiSanto JP, Muller W, Guy-Grand D, et al. Lymphoid development in mice with a targeted deletion of the interleukin 2 receptor-chain. Proc Natl Acad Sci U S A. 1995;92:277-281.
    • (1995) Proc Natl Acad Sci U S A , vol.92 , pp. 277-281
    • DiSanto, J.P.1    Muller, W.2    Guy-Grand, D.3
  • 40
    • 10544244162 scopus 로고    scopus 로고
    • Atypical X-linked severe combined immunodeficiency due to possible spontaneous reversion of the genetic defect in T cells
    • Stephan V, Wahn V, Le Deist F, et al. Atypical X-linked severe combined immunodeficiency due to possible spontaneous reversion of the genetic defect in T cells. N Engl J Med. 1996;335:1563-1567.
    • (1996) N Engl J Med , vol.335 , pp. 1563-1567
    • Stephan, V.1    Wahn, V.2    Le Deist, F.3
  • 41
    • 0142084745 scopus 로고    scopus 로고
    • LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCIDX1
    • Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCIDX1. Science. 2003;302:415- 419.
    • (2003) Science , vol.302 , pp. 415-419
    • Hacein-Bey-Abina, S.1    Von Kalle, C.2    Schmidt, M.3
  • 42
    • 0033118161 scopus 로고    scopus 로고
    • The effect of chromosomal translocations in acute leukemias: The LMO2 paradigm in transcription and development
    • Rabbitts TH, Bucher K, Chung G, et al. The effect of chromosomal translocations in acute leukemias: the LMO2 paradigm in transcription and development. Cancer Res. 1999;59:1794s-1798s.
    • (1999) Cancer Res , vol.59
    • Rabbitts, T.H.1    Bucher, K.2    Chung, G.3
  • 43
    • 38149065392 scopus 로고    scopus 로고
    • Cole A. Child in gene therapy programme develops leukaemia. BMJ. 2008;336:13.
    • Cole A. Child in gene therapy programme develops leukaemia. BMJ. 2008;336:13.
  • 44
    • 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
  • 45
    • 85031347043 scopus 로고    scopus 로고
    • Stem cell gene therapy to treat X-linked severe combined immunodeficiency (XSCID). Clinicaltrials.gov Web site. http://www.clinical trials.gov/ct2/show/ NCT00028236?term-gene-therapy&rank-22. Accessed June 22, 2008.
    • Stem cell gene therapy to treat X-linked severe combined immunodeficiency (XSCID). Clinicaltrials.gov Web site. http://www.clinical trials.gov/ct2/show/ NCT00028236?term-gene-therapy&rank-22. Accessed June 22, 2008.
  • 46
    • 1342289322 scopus 로고    scopus 로고
    • Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency
    • McCormack MP, Rabbitts TH. Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2004;350:913-922.
    • (2004) N Engl J Med , vol.350 , pp. 913-922
    • McCormack, M.P.1    Rabbitts, T.H.2
  • 47
    • 0142089747 scopus 로고    scopus 로고
    • Fatal systemic inflammatory response syndrome in an ornithine transcarbamylase deficient patient following adenoviral gene transfer
    • Raper SE, Chirmule N, Lee FS, et al. Fatal systemic inflammatory response syndrome in an ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab. 2003;80:148-158.
    • (2003) Mol Genet Metab , vol.80 , pp. 148-158
    • Raper, S.E.1    Chirmule, N.2    Lee, F.S.3
  • 48
    • 0037841763 scopus 로고    scopus 로고
    • Transcription start regions in the human genome are favored targets for MLV integration
    • Wu X, Li Y, Crise B, Burgess SM. Transcription start regions in the human genome are favored targets for MLV integration. Science. 2003; 300:1749-1751.
    • (2003) Science , vol.300 , pp. 1749-1751
    • Wu, X.1    Li, Y.2    Crise, B.3    Burgess, S.M.4
  • 49
    • 0347634456 scopus 로고    scopus 로고
    • Gene therapy insertional mutagenesis insights
    • Dave UP, Jenkins NA, Copeland NG. Gene therapy insertional mutagenesis insights. Science. 2004;303:333.
    • (2004) Science , vol.303 , pp. 333
    • Dave, U.P.1    Jenkins, N.A.2    Copeland, N.G.3
  • 50
    • 44349158394 scopus 로고    scopus 로고
    • DNA bar coding and pyrosequencing to analyze adverse events of therapeutic gene transfer
    • Wang GP, Garrigue A, Ciuffi A, et al. DNA bar coding and pyrosequencing to analyze adverse events of therapeutic gene transfer. Nucleic Acids Res. 2008;36:e49.
    • (2008) Nucleic Acids Res , vol.36
    • Wang, G.P.1    Garrigue, A.2    Ciuffi, A.3
  • 51
    • 0034255145 scopus 로고    scopus 로고
    • A chromatin insulator protects retrovirus vectors from chromosomal position effects
    • Emery DW, Yannaki E, Tubb J, et al. A chromatin insulator protects retrovirus vectors from chromosomal position effects. Proc Natl AcadSci U S A. 2000;97:9150-9155.
    • (2000) Proc Natl AcadSci U S A , vol.97 , pp. 9150-9155
    • Emery, D.W.1    Yannaki, E.2    Tubb, J.3
  • 52
    • 0037058955 scopus 로고    scopus 로고
    • The insulation of genes from external enhances and silencing chromatin
    • Burgess-Beusse B, Farrell C, Gaszner M, et al. The insulation of genes from external enhances and silencing chromatin. Proc Natl Acad Sci U S A. 2002;99(suppl 4):16433-16437.
    • (2002) Proc Natl Acad Sci U S A , vol.99 , Issue.SUPPL. 4 , pp. 16433-16437
    • Burgess-Beusse, B.1    Farrell, C.2    Gaszner, M.3
  • 53
    • 59449098985 scopus 로고    scopus 로고
    • Gene therapy for immunodeficiency due to adenosine deaminase deficiency
    • Aiuti A, Cattaneo F, Galimberti S, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Eng J Med. 2009; 360:447- 458.
    • (2009) N Eng J Med , vol.360 , pp. 447-458
    • Aiuti, A.1    Cattaneo, F.2    Galimberti, S.3
  • 54
    • 59449108477 scopus 로고    scopus 로고
    • Gene therapy fulfilling its promise
    • Kohn DB, Candotti F. Gene therapy fulfilling its promise. N Eng J Med. 2009;360:518-521.
    • (2009) N Eng J Med , vol.360 , pp. 518-521
    • Kohn, D.B.1    Candotti, F.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.