메뉴 건너뛰기




Volumn 22, Issue 8, 2014, Pages 1472-1483

Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis

Author keywords

[No Author keywords available]

Indexed keywords

LENTIVIRUS VECTOR; MICRORNA 126; REACTIVE OXYGEN METABOLITE; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE 2; CD34 ANTIGEN; CYBB PROTEIN, HUMAN; MEMBRANE PROTEIN; MICRORNA; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE PHOSPHATE OXIDASE;

EID: 84905451458     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.87     Document Type: Article
Times cited : (57)

References (46)
  • 1
    • 75949110589 scopus 로고    scopus 로고
    • Chronic granulomatous disease
    • Holland, SM (2010). Chronic granulomatous disease. Clin Rev Allergy Immunol 38: 3-10.
    • (2010) Clin Rev Allergy Immunol , vol.38 , pp. 3-10
    • Holland, S.M.1
  • 2
    • 78649790896 scopus 로고    scopus 로고
    • Chronic granulomatous disease: Recent advances in pathophysiology and treatment
    • Seger, RA (2010). Chronic granulomatous disease: recent advances in pathophysiology and treatment. Neth J Med 68: 334-340.
    • (2010) Neth J Med , vol.68 , pp. 334-340
    • Seger, R.A.1
  • 3
    • 65549083114 scopus 로고    scopus 로고
    • Molecular characterization of a large cohort of patients with Chronic Granulomatous Disease and identification of novel CYBB mutations: An Italian multicenter study
    • IPINET (Italian Network for Primary Immunodeficiencies)
    • Di Matteo, G, Giordani, L, Finocchi, A, Ventura, A, Chiriaco, M, Blancato, J et al. ; IPINET (Italian Network for Primary Immunodeficiencies). (2009). Molecular characterization of a large cohort of patients with Chronic Granulomatous Disease and identification of novel CYBB mutations: an Italian multicenter study. Mol Immunol 46: 1935-1941.
    • (2009) Mol Immunol , vol.46 , pp. 1935-1941
    • Di Matteo, G.1    Giordani, L.2    Finocchi, A.3    Ventura, A.4    Chiriaco, M.5    Blancato, J.6
  • 4
    • 77950299675 scopus 로고    scopus 로고
    • Hematologically important mutations: The autosomal recessive forms of chronic granulomatous disease (second update)
    • Roos, D, Kuhns, DB, Maddalena, A, Bustamante, J, Kannengiesser, C, de Boer, M et al. (2010). Hematologically important mutations: the autosomal recessive forms of chronic granulomatous disease (second update). Blood Cells Mol Dis 44: 291-299.
    • (2010) Blood Cells Mol Dis , vol.44 , pp. 291-299
    • Roos, D.1    Kuhns, D.B.2    Maddalena, A.3    Bustamante, J.4    Kannengiesser, C.5    De Boer, M.6
  • 5
    • 38349105032 scopus 로고    scopus 로고
    • Modern management of chronic granulomatous disease
    • Seger, RA (2008). Modern management of chronic granulomatous disease. Br J Haematol 140: 255-266.
    • (2008) Br J Haematol , vol.140 , pp. 255-266
    • Seger, R.A.1
  • 6
    • 69949184405 scopus 로고    scopus 로고
    • Strategies to modulate immune responses: A new frontier for gene therapy
    • Arruda, VR, Favaro, P and Finn, JD (2009). Strategies to modulate immune responses: a new frontier for gene therapy. Mol Ther 17: 1492-1503.
    • (2009) Mol Ther , vol.17 , pp. 1492-1503
    • Arruda, V.R.1    Favaro, P.2    Finn, J.D.3
  • 7
    • 84455205615 scopus 로고    scopus 로고
    • Excellent survival after sibling or unrelated donor stem cell transplantation for chronic granulomatous disease
    • Martinez, CA, Shah, S, Shearer, WT, Rosenblatt, HM, Paul, ME, Chinen, J et al. (2012). Excellent survival after sibling or unrelated donor stem cell transplantation for chronic granulomatous disease. J Allergy Clin Immunol 129: 176-183.
    • (2012) J Allergy Clin Immunol , vol.129 , pp. 176-183
    • Martinez, C.A.1    Shah, S.2    Shearer, W.T.3    Rosenblatt, H.M.4    Paul, M.E.5    Chinen, J.6
  • 9
    • 77956395504 scopus 로고    scopus 로고
    • Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?
    • Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation; European Society for Immunodeficiency e1
    • Gennery, AR, Slatter, MA, Grandin, L, Taupin, P, Cant, AJ, Veys, P et al. ; Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation; European Society for Immunodeficiency. (2010). Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? J Allergy Clin Immunol 126: 602-10. e1.
    • (2010) J Allergy Clin Immunol , vol.126 , pp. 602-610
    • Gennery, A.R.1    Slatter, M.A.2    Grandin, L.3    Taupin, P.4    Cant, A.J.5    Veys, P.6
  • 10
    • 84895068883 scopus 로고    scopus 로고
    • Reduced-intensity conditioning and HLA-matched haemopoietic stemcell transplantation in patients with chronic granulomatous disease: A prospective multicentre study
    • Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation.
    • Güngör, T, Teira, P, Slatter, M, Stussi, G, Stepensky, P, Moshous, D et al. ; Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation. (2014). Reduced-intensity conditioning and HLA-matched haemopoietic stemcell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet 383: 436-448.
    • (2014) Lancet , vol.383 , pp. 436-448
    • Güngör, T.1    Teira, P.2    Slatter, M.3    Stussi, G.4    Stepensky, P.5    Moshous, D.6
  • 11
    • 77949327872 scopus 로고    scopus 로고
    • Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils
    • Kang, EM, Choi, U, Theobald, N, Linton, G, Long Priel, DA, Kuhns, D et al. (2010). Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils. Blood 115: 783-791.
    • (2010) Blood , vol.115 , pp. 783-791
    • Kang, E.M.1    Choi, U.2    Theobald, N.3    Linton, G.4    Long Priel, D.A.5    Kuhns, D.6
  • 12
    • 80455174635 scopus 로고    scopus 로고
    • Retroviral gene therapy for X-linked chronic granulomatous disease: Results from phase I/II trial
    • Kang, HJ, Bartholomae, CC, Paruzynski, A, Arens, A, Kim, S, Yu, SS et al. (2011). Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial. Mol Ther 19: 2092-2101.
    • (2011) Mol Ther , vol.19 , pp. 2092-2101
    • Kang, H.J.1    Bartholomae, C.C.2    Paruzynski, A.3    Arens, A.4    Kim, S.5    Yu, S.S.6
  • 13
    • 33645734405 scopus 로고    scopus 로고
    • Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
    • Ott, MG, Schmidt, M, Schwarzwaelder, K, Stein, S, Siler, U, Koehl, U et al. (2006). Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 12: 401-409.
    • (2006) Nat Med , vol.12 , pp. 401-409
    • Ott, M.G.1    Schmidt, M.2    Schwarzwaelder, K.3    Stein, S.4    Siler, U.5    Koehl, U.6
  • 14
    • 76249131912 scopus 로고    scopus 로고
    • Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
    • Stein, S, Ott, MG, Schultze-Strasser, S, Jauch, A, Burwinkel, B, Kinner, A et al. (2010). Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 16: 198-204.
    • (2010) Nat Med , vol.16 , pp. 198-204
    • Stein, S.1    Ott, M.G.2    Schultze-Strasser, S.3    Jauch, A.4    Burwinkel, B.5    Kinner, A.6
  • 15
    • 84255196304 scopus 로고    scopus 로고
    • Stem cell gene therapy: The risks of insertional mutagenesis and approaches to minimize genotoxicity
    • Wu, C and Dunbar, CE (2011). Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity. Front Med 5: 356-371.
    • (2011) Front Med , vol.5 , pp. 356-371
    • Wu, C.1    Dunbar, C.E.2
  • 16
    • 33846794822 scopus 로고    scopus 로고
    • The NOX family of ROS-generating NADPH oxidases: Physiology and pathophysiology
    • Bedard, K and Krause, KH (2007). The NOX family of ROS-generating NADPH oxidases: physiology and pathophysiology. Physiol Rev 87: 245-313.
    • (2007) Physiol Rev , vol.87 , pp. 245-313
    • Bedard, K.1    Krause, K.H.2
  • 17
    • 80052930073 scopus 로고    scopus 로고
    • Accumulation of oxidative DNA damage restricts the self-renewal capacity of human hematopoietic stem cells
    • Yahata, T, Takanashi, T, Muguruma, Y, Ibrahim, AA, Matsuzawa, H, Uno, T et al. (2011). Accumulation of oxidative DNA damage restricts the self-renewal capacity of human hematopoietic stem cells. Blood 118: 2941-2950.
    • (2011) Blood , vol.118 , pp. 2941-2950
    • Yahata, T.1    Takanashi, T.2    Muguruma, Y.3    Ibrahim, A.A.4    Matsuzawa, H.5    Uno, T.6
  • 19
    • 79954692758 scopus 로고    scopus 로고
    • Ex vivo gene transfer and correction for cell-based therapies
    • Naldini, L (2011). Ex vivo gene transfer and correction for cell-based therapies. Nat Rev Genet 12: 301-315.
    • (2011) Nat Rev Genet , vol.12 , pp. 301-315
    • Naldini, L.1
  • 20
    • 78650918644 scopus 로고    scopus 로고
    • Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells
    • Santilli, G, Almarza, E, Brendel, C, Choi, U, Beilin, C, Blundell, MP et al. (2011). Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells. Mol Ther 19: 122-132.
    • (2011) Mol Ther , vol.19 , pp. 122-132
    • Santilli, G.1    Almarza, E.2    Brendel, C.3    Choi, U.4    Beilin, C.5    Blundell, M.P.6
  • 22
    • 84867402831 scopus 로고    scopus 로고
    • Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter
    • Brendel, C, Müller-Kuller, U, Schultze-Strasser, S, Stein, S, Chen-Wichmann, L, Krattenmacher, A et al. (2012). Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter. Gene Ther 19: 1018-1029.
    • (2012) Gene Ther , vol.19 , pp. 1018-1029
    • Brendel, C.1    Müller-Kuller, U.2    Schultze-Strasser, S.3    Stein, S.4    Chen-Wichmann, L.5    Krattenmacher, A.6
  • 23
    • 80855132872 scopus 로고    scopus 로고
    • Lineage- and stage-restricted lentiviral vectors for the gene therapy of chronic granulomatous disease
    • Barde, I, Laurenti, E, Verp, S, Wiznerowicz, M, Offner, S, Viornery, A et al. (2011). Lineage- and stage-restricted lentiviral vectors for the gene therapy of chronic granulomatous disease. Gene Ther 18: 1087-1097.
    • (2011) Gene Ther , vol.18 , pp. 1087-1097
    • Barde, I.1    Laurenti, E.2    Verp, S.3    Wiznerowicz, M.4    Offner, S.5    Viornery, A.6
  • 24
    • 36849060584 scopus 로고    scopus 로고
    • Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
    • Brown, BD, Gentner, B, Cantore, A, Colleoni, S, Amendola, M, Zingale, A et al. (2007). Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol 25: 1457-1467.
    • (2007) Nat Biotechnol , vol.25 , pp. 1457-1467
    • Brown, B.D.1    Gentner, B.2    Cantore, A.3    Colleoni, S.4    Amendola, M.5    Zingale, A.6
  • 25
    • 33646564701 scopus 로고    scopus 로고
    • Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer
    • Brown, BD, Venneri, MA, Zingale, A, Sergi Sergi, L and Naldini, L (2006). Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med 12: 585-591.
    • (2006) Nat Med , vol.12 , pp. 585-591
    • Brown, B.D.1    Venneri, M.A.2    Zingale, A.3    Sergi Sergi, L.4    Naldini, L.5
  • 26
    • 84866869441 scopus 로고    scopus 로고
    • Exploiting microRNA regulation for genetic engineering
    • Gentner, B and Naldini, L (2012). Exploiting microRNA regulation for genetic engineering. Tissue Antigens 80: 393-403.
    • (2012) Tissue Antigens , vol.80 , pp. 393-403
    • Gentner, B.1    Naldini, L.2
  • 27
    • 78449270317 scopus 로고    scopus 로고
    • Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy
    • Gentner, B, Visigalli, I, Hiramatsu, H, Lechman, E, Ungari, S, Giustacchini, A et al. (2010). Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med 2: 58ra84.
    • (2010) Sci Transl Med , vol.2
    • Gentner, B.1    Visigalli, I.2    Hiramatsu, H.3    Lechman, E.4    Ungari, S.5    Giustacchini, A.6
  • 28
    • 33750608242 scopus 로고    scopus 로고
    • Development of a synthetic promoter for macrophage gene therapy
    • He, W, Qiang, M, Ma, W, Valente, AJ, Quinones, MP, Wang, W et al. (2006). Development of a synthetic promoter for macrophage gene therapy. Hum Gene Ther 17: 949-959.
    • (2006) Hum Gene Ther , vol.17 , pp. 949-959
    • He, W.1    Qiang, M.2    Ma, W.3    Valente, A.J.4    Quinones, M.P.5    Wang, W.6
  • 29
    • 58149469932 scopus 로고    scopus 로고
    • Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells
    • Moreno-Carranza, B, Gentsch, M, Stein, S, Schambach, A, Santilli, G, Rudolf, E et al. (2009). Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells. Gene Ther 16: 111-118.
    • (2009) Gene Ther , vol.16 , pp. 111-118
    • Moreno-Carranza, B.1    Gentsch, M.2    Stein, S.3    Schambach, A.4    Santilli, G.5    Rudolf, E.6
  • 30
    • 79958172300 scopus 로고    scopus 로고
    • A simple, versatile and efficient method to genetically modify human monocytederived dendritic cells with HIV-1-derived lentiviral vectors
    • Berger, G, Durand, S, Goujon, C, Nguyen, XN, Cordeil, S, Darlix, JL et al. (2011). A simple, versatile and efficient method to genetically modify human monocytederived dendritic cells with HIV-1-derived lentiviral vectors. Nat Protoc 6: 806-816.
    • (2011) Nat Protoc , vol.6 , pp. 806-816
    • Berger, G.1    Durand, S.2    Goujon, C.3    Nguyen, X.N.4    Cordeil, S.5    Darlix, J.L.6
  • 31
    • 79959858243 scopus 로고    scopus 로고
    • Vpx relieves inhibition of HIV-1 infection of macrophages mediated by the SAMHD1 protein
    • Hrecka, K, Hao, C, Gierszewska, M, Swanson, SK, Kesik-Brodacka, M, Srivastava, S et al. (2011). Vpx relieves inhibition of HIV-1 infection of macrophages mediated by the SAMHD1 protein. Nature 474: 658-661.
    • (2011) Nature , vol.474 , pp. 658-661
    • Hrecka, K.1    Hao, C.2    Gierszewska, M.3    Swanson, S.K.4    Kesik-Brodacka, M.5    Srivastava, S.6
  • 32
    • 48549106378 scopus 로고    scopus 로고
    • The endothelial-specific microRNA miR-126 governs vascular integrity and angiogenesis
    • Wang, S, Aurora, AB, Johnson, BA, Qi, X, McAnally, J, Hill, JA et al. (2008). The endothelial-specific microRNA miR-126 governs vascular integrity and angiogenesis. Dev Cell 15: 261-271.
    • (2008) Dev Cell , vol.15 , pp. 261-271
    • Wang, S.1    Aurora, A.B.2    Johnson, B.A.3    Qi, X.4    McAnally, J.5    Hill, J.A.6
  • 34
    • 55749099505 scopus 로고    scopus 로고
    • Distinct microRNA expression profiles in acute myeloid leukemia with common translocations
    • Li, Z, Lu, J, Sun, M, Mi, S, Zhang, H, Luo, RT et al. (2008). Distinct microRNA expression profiles in acute myeloid leukemia with common translocations. Proc Natl Acad Sci USA 105: 15535-15540.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 15535-15540
    • Li, Z.1    Lu, J.2    Sun, M.3    Mi, S.4    Zhang, H.5    Luo, R.T.6
  • 36
    • 77956265818 scopus 로고    scopus 로고
    • A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors
    • Zhang, F, Frost, AR, Blundell, MP, Bales, O, Antoniou, MN and Thrasher, AJ (2010). A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors. Mol Ther 18: 1640-1649.
    • (2010) Mol Ther , vol.18 , pp. 1640-1649
    • Zhang, F.1    Frost, A.R.2    Blundell, M.P.3    Bales, O.4    Antoniou, M.N.5    Thrasher, A.J.6
  • 37
    • 1842370351 scopus 로고    scopus 로고
    • Retroviral-mediated gene transfer of gp91phox into bone marrow cells rescues defect in host defense against Aspergillus fumigatus in murine X-linked chronic granulomatous disease
    • Björgvinsdóttir, H, Ding, C, Pech, N, Gifford, MA, Li, LL and Dinauer, MC (1997). Retroviral-mediated gene transfer of gp91phox into bone marrow cells rescues defect in host defense against Aspergillus fumigatus in murine X-linked chronic granulomatous disease. Blood 89: 41-48.
    • (1997) Blood , vol.89 , pp. 41-48
    • Björgvinsdóttir, H.1    Ding, C.2    Pech, N.3    Gifford, M.A.4    Li, L.L.5    Dinauer, M.C.6
  • 38
    • 84871929094 scopus 로고    scopus 로고
    • Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome
    • Scaramuzza, S, Biasco, L, Ripamonti, A, Castiello, MC, Loperfido, M, Draghici, E et al. (2013). Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome. Mol Ther 21: 175-184.
    • (2013) Mol Ther , vol.21 , pp. 175-184
    • Scaramuzza, S.1    Biasco, L.2    Ripamonti, A.3    Castiello, M.C.4    Loperfido, M.5    Draghici, E.6
  • 39
    • 69949164575 scopus 로고    scopus 로고
    • Cell-intrinsic and vector-related properties cooperate to determine the incidence and consequences of insertional mutagenesis
    • Kustikova, OS, Schiedlmeier, B, Brugman, MH, Stahlhut, M, Bartels, S, Li, Z et al. (2009). Cell-intrinsic and vector-related properties cooperate to determine the incidence and consequences of insertional mutagenesis. Mol Ther 17: 1537-1547.
    • (2009) Mol Ther , vol.17 , pp. 1537-1547
    • Kustikova, O.S.1    Schiedlmeier, B.2    Brugman, M.H.3    Stahlhut, M.4    Bartels, S.5    Li, Z.6
  • 40
    • 84857560044 scopus 로고    scopus 로고
    • Genotoxicity assay for gene therapy vectors in tumor prone Cdkn2a-/- mice
    • Montini, E and Cesana, D (2012). Genotoxicity assay for gene therapy vectors in tumor prone Cdkn2a-/- mice. Methods Enzymol 507: 171-185.
    • (2012) Methods Enzymol , vol.507 , pp. 171-185
    • Montini, E.1    Cesana, D.2
  • 41
    • 33750624015 scopus 로고    scopus 로고
    • Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity
    • Modlich, U, Bohne, J, Schmidt, M, von Kalle, C, Knöss, S, Schambach, A et al. (2006). Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood 108: 2545-2553.
    • (2006) Blood , vol.108 , pp. 2545-2553
    • Modlich, U.1    Bohne, J.2    Schmidt, M.3    Von Kalle, C.4    Knöss, S.5    Schambach, A.6
  • 42
    • 84879873039 scopus 로고    scopus 로고
    • Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
    • Biffi, A, Montini, E, Lorioli, L, Cesani, M, Fumagalli, F, Plati, T et al. (2013). Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341: 1233158.
    • (2013) Science , vol.341 , pp. 1233158
    • Biffi, A.1    Montini, E.2    Lorioli, L.3    Cesani, M.4    Fumagalli, F.5    Plati, T.6
  • 43
    • 84879867061 scopus 로고    scopus 로고
    • Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
    • Aiuti, A, Biasco, L, Scaramuzza, S, Ferrua, F, Cicalese, MP, Baricordi, C et al. (2013). Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 341: 1233151.
    • (2013) Science , vol.341 , pp. 1233151
    • Aiuti, A.1    Biasco, L.2    Scaramuzza, S.3    Ferrua, F.4    Cicalese, M.P.5    Baricordi, C.6
  • 46
    • 0025150592 scopus 로고
    • Kinetic microplate assay for superoxide production by neutrophils and other phagocytic cells
    • Mayo, LA and Curnutte, JT (1990). Kinetic microplate assay for superoxide production by neutrophils and other phagocytic cells. Methods Enzymol 186: 567-575.
    • (1990) Methods Enzymol , vol.186 , pp. 567-575
    • Mayo, L.A.1    Curnutte, J.T.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.