메뉴 건너뛰기




Volumn 101, Issue 7, 2003, Pages 2563-2569

Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: Long-term results of the first clinical gene therapy trial

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; COMPLEMENTARY DNA; MACROGOL; OKT 3; RECOMBINANT ENZYME; RETROVIRUS VECTOR; VIRUS ANTIBODY; HETEROPHILE ANTIBODY; LYMPHOCYTE ANTIGEN RECEPTOR; PEGADEMASE BOVINE;

EID: 0038446699     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood-2002-09-2800     Document Type: Article
Times cited : (193)

References (31)
  • 1
    • 0000930996 scopus 로고    scopus 로고
    • Immunodeficiency disease due to deficiency of adenosine deaminase
    • Ochs HD, Smith CIE, Puck JM, eds. New York, NY: Oxford University Press
    • Hirschhorn R. Immunodeficiency disease due to deficiency of adenosine deaminase. In: Ochs HD, Smith CIE, Puck JM, eds. Primary Immunodeficiency Diseases: A Molecular and Genetic Approach. New York, NY: Oxford University Press; 1999:121-139.
    • (1999) Primary Immunodeficiency Diseases: A Molecular and Genetic Approach , pp. 121-139
    • Hirschhorn, R.1
  • 2
    • 0000124054 scopus 로고    scopus 로고
    • Immunodeficiency diseases caused by adenosine deaminase deficiency ciency and purine nucleoside phosphorylase deficiency
    • Scriver CR, Beaudet AL, Sly WS, Valle D, eds. New York, NY: McGraw-Hill
    • Hershfield MS, Mitchell BS. Immunodeficiency diseases caused by adenosine deaminase deficiency ciency and purine nucleoside phosphorylase deficiency. In: Scriver CR, Beaudet AL, Sly WS, Valle D, eds. The Metabolic and Molecular Bases of Inherited Disease. New York, NY: McGraw-Hill; 2001:2585-2625.
    • (2001) The Metabolic and Molecular Bases of Inherited Disease , pp. 2585-2625
    • Hershfield, M.S.1    Mitchell, B.S.2
  • 3
    • 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
  • 4
    • 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
  • 5
    • 0023146216 scopus 로고
    • Treatment of adenosine deaminase deficiency with polyethylene glycol-modified adenosine deaminase
    • Hershfield MS, Buckley RH, Greenberg ML, et al. Treatment of adenosine deaminase deficiency with polyethylene glycol-modified adenosine deaminase. N Engl J Med. 1987;316:589-596.
    • (1987) N Engl J Med , vol.316 , pp. 589-596
    • Hershfield, M.S.1    Buckley, R.H.2    Greenberg, M.L.3
  • 6
    • 0019196862 scopus 로고
    • Erythrocyte disorders of purine and pyrimidine metabolism
    • Valentine WN, Paglia DE. Erythrocyte disorders of purine and pyrimidine metabolism. Hemoglobin. 1980;4:669-681.
    • (1980) Hemoglobin , vol.4 , pp. 669-681
    • Valentine, W.N.1    Paglia, D.E.2
  • 8
    • 0024392134 scopus 로고
    • Expression of human adenosine deaminase from various strong promoters after gene transfer into human hematopoietic cell lines
    • Hock RA, Miller AD, Osborne WR. Expression of human adenosine deaminase from various strong promoters after gene transfer into human hematopoietic cell lines. Blood. 1989;74:876-881.
    • (1989) Blood , vol.74 , pp. 876-881
    • Hock, R.A.1    Miller, A.D.2    Osborne, W.R.3
  • 9
    • 0028807727 scopus 로고
    • T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years
    • Blaese RM, Culver, KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science. 1995;270:475-480.
    • (1995) Science , vol.270 , pp. 475-480
    • Blaese, R.M.1    Culver, K.W.2    Miller, A.D.3
  • 10
    • 0029945161 scopus 로고    scopus 로고
    • Molecular analysis of T lymphocyte-directed gene therapy for adenosine deaminase deficiency: Long-term expression in vivo of genes introduced with a retroviral vector
    • Mullen CA, Snitzer K, Culver KW, Morgan RA, Anderson WF, Blaese RM. Molecular analysis of T lymphocyte-directed gene therapy for adenosine deaminase deficiency: long-term expression in vivo of genes introduced with a retroviral vector. Hum Gene Ther. 1996;7:1123-1129.
    • (1996) Hum Gene Ther , vol.7 , pp. 1123-1129
    • Mullen, C.A.1    Snitzer, K.2    Culver, K.W.3    Morgan, R.A.4    Anderson, W.F.5    Blaese, R.M.6
  • 11
    • 0023218174 scopus 로고
    • Retroviral-mediated gene transfer into mammalian cells
    • Kohn DB, Kantoff PW, Eglitis MA, et al. Retroviral-mediated gene transfer into mammalian cells. Blood Cells. 1987;13:285-298.
    • (1987) Blood Cells , vol.13 , pp. 285-298
    • Kohn, D.B.1    Kantoff, P.W.2    Eglitis, M.A.3
  • 12
    • 0028834086 scopus 로고
    • Oligonucleotides with fluorescent dyes at opposite ends provide a quenched probe system useful for detecting PCR product and nucleic acid hybridization
    • Livak KJ, Flood SJ, Marmaro J, Giusti W, Deetz K. Oligonucleotides with fluorescent dyes at opposite ends provide a quenched probe system useful for detecting PCR product and nucleic acid hybridization. PCR Methods Appl. 1995;4:357-362.
    • (1995) PCR Methods Appl , vol.4 , pp. 357-362
    • Livak, K.J.1    Flood, S.J.2    Marmaro, J.3    Giusti, W.4    Deetz, K.5
  • 13
    • 0035902552 scopus 로고    scopus 로고
    • Somatic mosaicism in Wiskott-Aldrich syndrome suggests in vivo reversion by a DNA slippage mechanism
    • 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 U S A , vol.98 , pp. 8697-8702
    • Wada, T.1    Schurman, S.H.2    Otsu, M.3
  • 14
    • 0024317148 scopus 로고
    • Establishment and characterization of adenosine deaminase-deficient human T cell lines
    • Kohn DB, Mitsuya H, Ballow M, et al. Establishment and characterization of adenosine deaminase-deficient human T cell lines. J Immunol. 1989;142:3971-3977.
    • (1989) J Immunol , vol.142 , pp. 3971-3977
    • Kohn, D.B.1    Mitsuya, H.2    Ballow, M.3
  • 15
    • 12344249949 scopus 로고
    • Amphotropic murine leukemia retrovirus is not an acute pathogen for primates
    • Cornetta K, Moen RC, Culver K, et al. Amphotropic murine leukemia retrovirus is not an acute pathogen for primates. Hum Gene Ther. 1990;1:15-30.
    • (1990) Hum Gene Ther , vol.1 , pp. 15-30
    • Cornetta, K.1    Moen, R.C.2    Culver, K.3
  • 16
    • 0027728743 scopus 로고
    • + selected autologous peripheral blood cells transduced with a human ADA gene. Amendment to clinical research project, Project 90-C-195, January 10, 1992
    • + selected autologous peripheral blood cells transduced with a human ADA gene. Amendment to clinical research project, Project 90-C-195, January 10, 1992. Hum Gene Ther. 1993;4:521-527.
    • (1993) Hum Gene Ther , vol.4 , pp. 521-527
    • Blaese, R.M.1    Culver, K.W.2    Chang, L.3
  • 17
    • 0027976958 scopus 로고
    • Clonal populations of T cells in normal elderly humans: The T cell equivalent to "benign monoclonal gammapathy"
    • Posnett DN, Sinha R, Kabak S, Russo C. Clonal populations of T cells in normal elderly humans: the T cell equivalent to "benign monoclonal gammapathy." J Exp Med. 1994;179:609-618.
    • (1994) J Exp Med , vol.179 , pp. 609-618
    • Posnett, D.N.1    Sinha, R.2    Kabak, S.3    Russo, C.4
  • 19
    • 0028920850 scopus 로고
    • + T cell expansions in normal individuals and patients with rheumatoid arthritis
    • + T cell expansions in normal individuals and patients with rheumatoid arthritis. J Immunol. 1995;154:3538-3547.
    • (1995) J Immunol , vol.154 , pp. 3538-3547
    • Fitzgerald, J.E.1    Ricalton, N.S.2    Meyer, A.C.3
  • 20
    • 0028220953 scopus 로고
    • Circulating T cell repertoire complexity in normal individuals and bone marrow recipients analyzed by CDR3 size spectratyping: Correlation with immune status
    • Gorski J, Yassai M, Zhu X, et al. Circulating T cell repertoire complexity in normal individuals and bone marrow recipients analyzed by CDR3 size spectratyping: correlation with immune status. J Immunol. 1994;152:5109-5119.
    • (1994) J Immunol , vol.152 , pp. 5109-5119
    • Gorski, J.1    Yassai, M.2    Zhu, X.3
  • 21
    • 0029913896 scopus 로고    scopus 로고
    • Transduction of pluripotent human hematopoietic stem cells demonstrated by clonal analysis after engraftment in immune-deficient mice
    • Nolta JA, Dao MA, Wells S, Smogorzewska EM, Kohn DB. Transduction of pluripotent human hematopoietic stem cells demonstrated by clonal analysis after engraftment in immune-deficient mice. Proc Natl Acad Sci U S A. 1996;93:2414-2419.
    • (1996) Proc Natl Acad Sci U S A , vol.93 , pp. 2414-2419
    • Nolta, J.A.1    Dao, M.A.2    Wells, S.3    Smogorzewska, E.M.4    Kohn, D.B.5
  • 22
    • 0014442965 scopus 로고
    • Micromodification of the Ouchterlony gel diffusion precipitin technique
    • Szatalowicz FT, Blenden DC, Parker CL. Micromodification of the Ouchterlony gel diffusion precipitin technique. Am J Vet Res. 1969;30:149-150.
    • (1969) Am J Vet Res , vol.30 , pp. 149-150
    • Szatalowicz, F.T.1    Blenden, D.C.2    Parker, C.L.3
  • 23
    • 0031975088 scopus 로고    scopus 로고
    • Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency
    • Onodera M, Ariga T, Kawamura N, et al. Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency. Blood. 1998;91:30-36.
    • (1998) Blood , vol.91 , pp. 30-36
    • Onodera, M.1    Ariga, T.2    Kawamura, N.3
  • 24
    • 0026212584 scopus 로고
    • No retroviremia or pathology in long-term follow-up of monkeys exposed to a murine amphotropic retrovirus
    • Cornetta K, Morgan RA, Gillio A, et al. No retroviremia or pathology in long-term follow-up of monkeys exposed to a murine amphotropic retrovirus. Hum Gene Ther. 1991;2:215-219.
    • (1991) Hum Gene Ther , vol.2 , pp. 215-219
    • Cornetta, K.1    Morgan, R.A.2    Gillio, A.3
  • 25
    • 17344372848 scopus 로고    scopus 로고
    • Biosafety monitoring of patients receiving intracerebral injections of murine retroviral vector producer cells
    • Long Z, Li LP, Grooms T, et al. Biosafety monitoring of patients receiving intracerebral injections of murine retroviral vector producer cells. Hum Gene Ther. 1998;9:1165-1172.
    • (1998) Hum Gene Ther , vol.9 , pp. 1165-1172
    • Long, Z.1    Li, L.P.2    Grooms, T.3
  • 26
    • 0023813205 scopus 로고
    • Adenosine deaminase deficiency with late onset of recurrent infections: Response to treatment with polyethylene glycol-modified adenosine deaminase
    • Levy Y, Hershfield MS, Fernandez-Mejia C, et al. Adenosine deaminase deficiency with late onset of recurrent infections: response to treatment with polyethylene glycol-modified adenosine deaminase. J Pediatr. 1988;113:312-317.
    • (1988) J Pediatr , vol.113 , pp. 312-317
    • Levy, Y.1    Hershfield, M.S.2    Fernandez-Mejia, C.3
  • 27
    • 0030892087 scopus 로고    scopus 로고
    • Development of antibodies to fetal calf serum with arthus-like reactions in human immunodeficiency virus-infected patients given syngeneic lymphocyte infusions
    • Selvaggi TA, Walker RE, Fleisher TA. Development of antibodies to fetal calf serum with arthus-like reactions in human immunodeficiency virus-infected patients given syngeneic lymphocyte infusions. Blood. 1997;89:776-779.
    • (1997) Blood , vol.89 , pp. 776-779
    • Selvaggi, T.A.1    Walker, R.E.2    Fleisher, T.A.3
  • 28
    • 13344261952 scopus 로고    scopus 로고
    • T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients
    • Riddell SR, Elliott M, Lewinsohn DA, et al. T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients. Nat Med. 1996;2:216-223.
    • (1996) Nat Med , vol.2 , pp. 216-223
    • Riddell, S.R.1    Elliott, M.2    Lewinsohn, D.A.3
  • 29
    • 0030843493 scopus 로고    scopus 로고
    • HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia
    • Bonini C, Ferrari G, Verzeletti S, et al. HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science. 1997;276:1719-1724.
    • (1997) Science , vol.276 , pp. 1719-1724
    • Bonini, C.1    Ferrari, G.2    Verzeletti, S.3
  • 30
    • 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
  • 31
    • 0037189401 scopus 로고    scopus 로고
    • Correction of ADA-SCID by stem cell gene therapy combined with non-myeloablative conditioning
    • Aiuti A, Slavin S, Aker M, et al. Correction of ADA-SCID by stem cell gene therapy combined with non-myeloablative conditioning. Science. 2002;296:2410-2413.
    • (2002) Science , vol.296 , pp. 2410-2413
    • Aiuti, A.1    Slavin, S.2    Aker, M.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.