메뉴 건너뛰기




Volumn 24, Issue 9, 2010, Pages 1617-1630

Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized human BTK

Author keywords

B cells; BTK; gene therapy; lentivirus; stem cells; XLA

Indexed keywords

B LYMPHOCYTE RECEPTOR; BRUTON TYROSINE KINASE; IMMUNOGLOBULIN G3; IMMUNOGLOBULIN M; LENTIVIRUS VECTOR; T LYMPHOCYTE ANTIGEN; AGAMMAGLOBULINAEMIA TYROSINE KINASE; CALCIUM; CODON; PRIMER DNA; PROTEIN TYROSINE KINASE;

EID: 77956428092     PISSN: 08876924     EISSN: 14765551     Source Type: Journal    
DOI: 10.1038/leu.2010.140     Document Type: Article
Times cited : (38)

References (58)
  • 1
    • 0345865076 scopus 로고    scopus 로고
    • Human primary immunodeficiency diseases: A perspective
    • Fischer A. Human primary immunodeficiency diseases: a perspective. Nat Immunol 2004; 5: 23-30.
    • (2004) Nat Immunol , vol.5 , pp. 23-30
    • Fischer, A.1
  • 2
    • 34249890562 scopus 로고    scopus 로고
    • Gene therapy for severe combined immunodeficiency: Are we there yet?
    • Cavazzana-Calvo M, Fischer A. Gene therapy for severe combined immunodeficiency: are we there yet? J Clin Invest 2007; 117: 1456-1465.
    • (2007) J Clin Invest , vol.117 , pp. 1456-1465
    • Cavazzana-Calvo, M.1    Fischer, A.2
  • 3
    • 35549013276 scopus 로고    scopus 로고
    • New insights and unresolved issues regarding insertional mutagenesis in X-linked SCID gene therapy
    • Pike-Overzet K, van der Burg M, Wagemaker G, van Dongen JJ, Staal FJ. New insights and unresolved issues regarding insertional mutagenesis in X-linked SCID gene therapy. Mol Ther 2007; 15: 1910-1916.
    • (2007) Mol Ther , vol.15 , pp. 1910-1916
    • Pike-Overzet, K.1    Van Der Burg, M.2    Wagemaker, G.3    Van Dongen, J.J.4    Staal, F.J.5
  • 4
    • 0027441332 scopus 로고
    • The gene involved in X-linked agammaglobulinaemia is a member of the src family of protein-tyrosine kinases
    • Vetrie D, Vorechovsky I, Sideras P, Holland J, Davies A, Flinter F et al. The gene involved in X-linked agammaglobulinaemia is a member of the src family of protein-tyrosine kinases. Nature 1993; 361: 226-233.
    • (1993) Nature , vol.361 , pp. 226-233
    • Vetrie, D.1    Vorechovsky, I.2    Sideras, P.3    Holland, J.4    Davies, A.5    Flinter, F.6
  • 5
    • 0027399081 scopus 로고
    • Deficient expression of a B cell cytoplasmic tyrosine kinase in human X-linked agammaglobulinemia
    • Tsukada S, Saffran DC, Rawlings DJ, Parolini O, Allen RC, Klisak I et al. Deficient expression of a B cell cytoplasmic tyrosine kinase in human X-linked agammaglobulinemia. Cell 1993; 72: 279-290.
    • (1993) Cell , vol.72 , pp. 279-290
    • Tsukada, S.1    Saffran, D.C.2    Rawlings, D.J.3    Parolini, O.4    Allen, R.C.5    Klisak, I.6
  • 6
    • 0001584969 scopus 로고    scopus 로고
    • The Tec family of cytoplasmic tyrosine kinases: Mammalian Btk, Bmx, Itk, Tec, Txk and homologs in other species
    • Smith CI, Islam TC, Mattsson PT, Mohamed AJ, Nore BF, Vihinen M. The Tec family of cytoplasmic tyrosine kinases: mammalian Btk, Bmx, Itk, Tec, Txk and homologs in other species. Bioessays 2001; 23: 436-446.
    • (2001) Bioessays , vol.23 , pp. 436-446
    • Smith, C.I.1    Islam, T.C.2    Mattsson, P.T.3    Mohamed, A.J.4    Nore, B.F.5    Vihinen, M.6
  • 7
    • 3843135096 scopus 로고    scopus 로고
    • Pleiotropic consequences of Bruton tyrosine kinase deficiency in myeloid lineages lead to poor inflammatory responses
    • Mangla A, Khare A, Vineeth V, Panday NN, Mukhopadhyay A, Ravindran B et al. Pleiotropic consequences of Bruton tyrosine kinase deficiency in myeloid lineages lead to poor inflammatory responses. Blood 2004; 104: 1191-1197.
    • (2004) Blood , vol.104 , pp. 1191-1197
    • Mangla, A.1    Khare, A.2    Vineeth, V.3    Panday, N.N.4    Mukhopadhyay, A.5    Ravindran, B.6
  • 9
    • 0043092190 scopus 로고    scopus 로고
    • Reduced dosage of Brutons tyrosine kinase uncouples B cell hyperresponsiveness from autoimmunity in lyn-/- mice
    • Whyburn LR, Halcomb KE, Contreras CM, Lowell CA, Witte ON, Satterthwaite AB. Reduced dosage of Brutons tyrosine kinase uncouples B cell hyperresponsiveness from autoimmunity in lyn-/- mice. J Immunol 2003; 171: 1850-1858.
    • (2003) J Immunol , vol.171 , pp. 1850-1858
    • Whyburn, L.R.1    Halcomb, K.E.2    Contreras, C.M.3    Lowell, C.A.4    Witte, O.N.5    Satterthwaite, A.B.6
  • 10
    • 0027358430 scopus 로고
    • The Brutons tyrosine kinase gene is expressed throughout B cell differentiation, from early precursor B cell stages preceding immunoglobulin gene rearrangement up to mature B cell stages
    • de Weers M, Verschuren MC, Kraakman ME, Mensink RG, Schuurman RK, van Dongen JJ et al. The Brutons tyrosine kinase gene is expressed throughout B cell differentiation, from early precursor B cell stages preceding immunoglobulin gene rearrangement up to mature B cell stages. Eur J Immunol 1993; 23: 3109-3114.
    • (1993) Eur J Immunol , vol.23 , pp. 3109-3114
    • De Weers, M.1    Verschuren, M.C.2    Kraakman, M.E.3    Mensink, R.G.4    Schuurman, R.K.5    Van Dongen, J.J.6
  • 11
    • 0028063861 scopus 로고
    • Mutation analysis of the Brutons tyrosine kinase gene in X-linked agammaglobulinemia: Identification of a mutation which affects the same codon as is altered in immunodeficient xid mice
    • de Weers M, Mensink RG, Kraakman ME, Schuurman RK, Hendriks RW. Mutation analysis of the Brutons tyrosine kinase gene in X-linked agammaglobulinemia: identification of a mutation which affects the same codon as is altered in immunodeficient xid mice. Hum Mol Genet 1994; 3: 161-166.
    • (1994) Hum Mol Genet , vol.3 , pp. 161-166
    • De Weers, M.1    Mensink, R.G.2    Kraakman, M.E.3    Schuurman, R.K.4    Hendriks, R.W.5
  • 12
    • 0028295482 scopus 로고
    • Expression of Brutons agammaglobulinemia tyrosine kinase gene, BTK, is selectively down-regulated in T lymphocytes and plasma cells
    • Smith CI, Baskin B, Humire-Greiff P, Zhou JN, Olsson PG, Maniar HS et al. Expression of Brutons agammaglobulinemia tyrosine kinase gene, BTK, is selectively down-regulated in T lymphocytes and plasma cells. J Immunol 1994; 152: 557-565.
    • (1994) J Immunol , vol.152 , pp. 557-565
    • Smith, C.I.1    Baskin, B.2    Humire-Greiff, P.3    Zhou, J.N.4    Olsson, P.G.5    Maniar, H.S.6
  • 13
    • 13144257712 scopus 로고    scopus 로고
    • Control of erythropoiesis by erythropoietin and stem cell factor: A novel role for Brutons tyrosine kinase
    • von Lindern M, Schmidt U, Beug H. Control of erythropoiesis by erythropoietin and stem cell factor: a novel role for Brutons tyrosine kinase. Cell Cycle 2004; 3: 876-879.
    • (2004) Cell Cycle , vol.3 , pp. 876-879
    • Von Lindern, M.1    Schmidt, U.2    Beug, H.3
  • 14
    • 0028314655 scopus 로고
    • Tyrosine phosphorylation of Blk and Fyn Src homology 2 domain-binding proteins occurs in response to antigen-receptor ligation in B cells and constitutively in pre-B cells
    • Aoki Y, Isselbacher KJ, Cherayil BJ, Pillai S. Tyrosine phosphorylation of Blk and Fyn Src homology 2 domain-binding proteins occurs in response to antigen-receptor ligation in B cells and constitutively in pre-B cells. Proceedings of the National Academy of Sciences of the United States of America 1994; 91: 4204-4208.
    • (1994) Proceedings of the National Academy of Sciences of the United States of America , vol.91 , pp. 4204-4208
    • Aoki, Y.1    Isselbacher, K.J.2    Cherayil, B.J.3    Pillai, S.4
  • 15
    • 0028151518 scopus 로고
    • Bruton tyrosine kinase is tyrosine phosphorylated and activated in pre-B lymphocytes and receptorligated B cells
    • Aoki Y, Isselbacher KJ, Pillai S. Bruton tyrosine kinase is tyrosine phosphorylated and activated in pre-B lymphocytes and receptorligated B cells. Proc Natl Acad Sci USA 1994; 91: 10606-10609.
    • (1994) Proc Natl Acad Sci USA , vol.91 , pp. 10606-10609
    • Aoki, Y.1    Isselbacher, K.J.2    Pillai, S.3
  • 16
    • 19944434293 scopus 로고    scopus 로고
    • Brutons tyrosine kinase: Cell biology, sequence conservation, mutation spectrum, siRNA modifications, and expression profiling
    • Lindvall JM, Blomberg KE, Valiaho J, Vargas L, Heinonen JE, Berglof A et al. Brutons tyrosine kinase: cell biology, sequence conservation, mutation spectrum, siRNA modifications, and expression profiling. Immunol Rev 2005; 203: 200-215.
    • (2005) Immunol Rev , vol.203 , pp. 200-215
    • Lindvall, J.M.1    Blomberg, K.E.2    Valiaho, J.3    Vargas, L.4    Heinonen, J.E.5    Berglof, A.6
  • 18
    • 40749083241 scopus 로고    scopus 로고
    • Gross deletions involving IGHM, BTK, or Artemis: A model for genomic lesions mediated by transposable elements
    • van Zelm MC, Geertsema C, Nieuwenhuis N, de Ridder D, Conley ME, Schiff C et al. Gross deletions involving IGHM, BTK, or Artemis: a model for genomic lesions mediated by transposable elements. Am J Hum Genet 2008; 82: 320-332.
    • (2008) Am J Hum Genet , vol.82 , pp. 320-332
    • Van Zelm, M.C.1    Geertsema, C.2    Nieuwenhuis, N.3    De Ridder, D.4    Conley, M.E.5    Schiff, C.6
  • 19
    • 0036152373 scopus 로고    scopus 로고
    • Composition of precursor B-cell compartment in bone marrow from patients with X-linked agammaglobulinemia compared with healthy children
    • Noordzij JG, de Bruin-Versteeg S, Comans-Bitter WM, Hartwig NG, Hendriks RW, de Groot R et al. Composition of precursor B-cell compartment in bone marrow from patients with X-linked agammaglobulinemia compared with healthy children. Pediatr Res 2002; 51: 159-168.
    • (2002) Pediatr Res , vol.51 , pp. 159-168
    • Noordzij, J.G.1    De Bruin-Versteeg, S.2    Comans-Bitter, W.M.3    Hartwig, N.G.4    Hendriks, R.W.5    De Groot, R.6
  • 21
    • 0022611188 scopus 로고
    • X-linked immune deficiency (xid) of CBA/N mice
    • Wicker LS, Scher I. X-linked immune deficiency (xid) of CBA/N mice. Curr Top Microbiol Immunol 1986; 124: 87-101.
    • (1986) Curr Top Microbiol Immunol , vol.124 , pp. 87-101
    • Wicker, L.S.1    Scher, I.2
  • 23
    • 0037087339 scopus 로고    scopus 로고
    • Impaired precursor B cell differentiation in Brutons tyrosine kinase-deficient mice
    • Middendorp S, Dingjan GM, Hendriks RW. Impaired precursor B cell differentiation in Brutons tyrosine kinase-deficient mice. J Immunol 2002; 168: 2695-2703.
    • (2002) J Immunol , vol.168 , pp. 2695-2703
    • Middendorp, S.1    Dingjan, G.M.2    Hendriks, R.W.3
  • 25
    • 4444311392 scopus 로고    scopus 로고
    • Sustained correction of B-cell development and function in a murine model of X-linked agammaglobulinemia (XLA) using retroviral-mediated gene transfer
    • Yu PW, Tabuchi RS, Kato RM, Astrakhan A, Humblet-Baron S, Kipp K et al. Sustained correction of B-cell development and function in a murine model of X-linked agammaglobulinemia (XLA) using retroviral-mediated gene transfer. Blood 2004; 104: 1281-1290.
    • (2004) Blood , vol.104 , pp. 1281-1290
    • Yu, P.W.1    Tabuchi, R.S.2    Kato, R.M.3    Astrakhan, A.4    Humblet-Baron, S.5    Kipp, K.6
  • 26
    • 0034606219 scopus 로고    scopus 로고
    • Severe B cell deficiency in mice lacking the tec kinase family members Tec and Btk
    • Ellmeier W, Jung S, Sunshine MJ, Hatam F, Xu Y, Baltimore D et al. Severe B cell deficiency in mice lacking the tec kinase family members Tec and Btk. J Exp Med 2000; 192: 1611-1624.
    • (2000) J Exp Med , vol.192 , pp. 1611-1624
    • Ellmeier, W.1    Jung, S.2    Sunshine, M.J.3    Hatam, F.4    Xu, Y.5    Baltimore, D.6
  • 27
    • 44849103042 scopus 로고    scopus 로고
    • Development of an enhanced B-specific lentiviral vector expressing BTK: A tool for gene therapy of XLA
    • Moreau T, Barlogis V, Bardin F, Nunes JA, Calmels B, Chabannon C et al. Development of an enhanced B-specific lentiviral vector expressing BTK: a tool for gene therapy of XLA. Gene Ther 2008; 15: 942-952.
    • (2008) Gene Ther , vol.15 , pp. 942-952
    • Moreau, T.1    Barlogis, V.2    Bardin, F.3    Nunes, J.A.4    Calmels, B.5    Chabannon, C.6
  • 30
    • 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, King D, Gilmour KC, Sinclair J 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    King, D.4    Gilmour, K.C.5    Sinclair, J.6
  • 31
    • 39849110286 scopus 로고    scopus 로고
    • Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency
    • Thornhill SI, Schambach A, Howe SJ, Ulaganathan M, Grassman E, Williams D et al. Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Mol Ther 2008; 16: 590-598.
    • (2008) Mol Ther , vol.16 , pp. 590-598
    • Thornhill, S.I.1    Schambach, A.2    Howe, S.J.3    Ulaganathan, M.4    Grassman, E.5    Williams, D.6
  • 32
    • 34249906359 scopus 로고    scopus 로고
    • Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy
    • Deichmann A, Hacein-Bey-Abina S, Schmidt M, Garrigue A, Brugman MH, Hu J et al. Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest 2007; 117: 2225-2232.
    • (2007) J Clin Invest , vol.117 , pp. 2225-2232
    • Deichmann, A.1    Hacein-Bey-Abina, S.2    Schmidt, M.3    Garrigue, A.4    Brugman, M.H.5    Hu, J.6
  • 34
    • 33745108790 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration
    • Montini E, Cesana D, Schmidt M, Sanvito F, Ponzoni M, Bartholomae C et al. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol 2006; 24: 687-696.
    • (2006) Nat Biotechnol , vol.24 , pp. 687-696
    • Montini, E.1    Cesana, D.2    Schmidt, M.3    Sanvito, F.4    Ponzoni, M.5    Bartholomae, C.6
  • 35
    • 0037162715 scopus 로고    scopus 로고
    • HIV-1 integration in the human genome favors active genes and local hotspots
    • Schroder AR, Shinn P, Chen H, Berry C, Ecker JR, Bushman F. HIV-1 integration in the human genome favors active genes and local hotspots. Cell 2002; 110: 521-529.
    • (2002) Cell , vol.110 , pp. 521-529
    • Schroder, A.R.1    Shinn, P.2    Chen, H.3    Berry, C.4    Ecker, J.R.5    Bushman, F.6
  • 36
    • 33746801652 scopus 로고    scopus 로고
    • Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy
    • Shou Y, Ma Z, Lu T, Sorrentino BP. Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy. Proc Natl Acad Sci USA 2006; 103: 11730-11735.
    • (2006) Proc Natl Acad Sci USA , vol.103 , pp. 11730-11735
    • Shou, Y.1    Ma, Z.2    Lu, T.3    Sorrentino, B.P.4
  • 37
    • 33750624015 scopus 로고    scopus 로고
    • Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity
    • Modlich U, Bohne J, SchmidtM, von Kalle C, Knoss S, Schambach A et al. Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood 2006; 108: 2545-2553.
    • (2006) Blood , vol.108 , pp. 2545-2553
    • Modlich, U.1    Bohne, J.2    Schmidtm Von Kalle, C.3    Knoss, S.4    Schambach, A.5
  • 38
    • 49449084991 scopus 로고    scopus 로고
    • Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16
    • Modlich U, Schambach A, Brugman MH, Wicke DC, Knoess S, Li Z et al. Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16. Leukemia 2008; 22: 1519-1528.
    • (2008) Leukemia , vol.22 , pp. 1519-1528
    • Modlich, U.1    Schambach, A.2    Brugman, M.H.3    Wicke, D.C.4    Knoess, S.5    Li, Z.6
  • 39
    • 0029792213 scopus 로고    scopus 로고
    • Inactivation of Btk by insertion of lacZ reveals defects in B cell development only past the pre-B cell stage
    • Hendriks RW, de Bruijn MF, Maas A, Dingjan GM, Karis A, Grosveld F. Inactivation of Btk by insertion of lacZ reveals defects in B cell development only past the pre-B cell stage. Embo J 1996; 15: 4862-4872.
    • (1996) Embo J , vol.15 , pp. 4862-4872
    • Hendriks, R.W.1    De Bruijn, M.F.2    Maas, A.3    Dingjan, G.M.4    Karis, A.5    Grosveld, F.6
  • 40
    • 34249298695 scopus 로고    scopus 로고
    • Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors
    • Schambach A, Galla M, Maetzig T, Loew R, Baum C. Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors. Mol Ther 2007; 15: 1167-1173.
    • (2007) Mol Ther , vol.15 , pp. 1167-1173
    • Schambach, A.1    Galla, M.2    Maetzig, T.3    Loew, R.4    Baum, C.5
  • 41
    • 3142704467 scopus 로고    scopus 로고
    • B-cell-specific transgene expression using a self-inactivating retroviral vector with human CD19 promoter and viral post-transcriptional regulatory element
    • Werner M, Kraunus J, Baum C, Brocker T. B-cell-specific transgene expression using a self-inactivating retroviral vector with human CD19 promoter and viral post-transcriptional regulatory element. Gene Ther 2004; 11: 992-1000.
    • (2004) Gene Ther , vol.11 , pp. 992-1000
    • Werner, M.1    Kraunus, J.2    Baum, C.3    Brocker, T.4
  • 42
    • 0030808926 scopus 로고    scopus 로고
    • The potent enhancer activity of the polycythemic strain of spleen focusforming virus in hematopoietic cells is governed by a binding site for Sp1 in the upstream control region and by a unique enhancer core motif, creating an exclusive target for PEBP/CBF
    • Baum C, Itoh K, Meyer J, Laker C, Ito Y, Ostertag W. The potent enhancer activity of the polycythemic strain of spleen focusforming virus in hematopoietic cells is governed by a binding site for Sp1 in the upstream control region and by a unique enhancer core motif, creating an exclusive target for PEBP/CBF. J virol 1997; 71: 6323-6331.
    • (1997) J Virol , vol.71 , pp. 6323-6331
    • Baum, C.1    Itoh, K.2    Meyer, J.3    Laker, C.4    Ito, Y.5    Ostertag, W.6
  • 43
    • 30344464717 scopus 로고    scopus 로고
    • Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells
    • Schambach A, Bohne J, Chandra S, Will E, Margison GP, Williams DA et al. Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. Mol Ther 2006; 13: 391-400.
    • (2006) Mol Ther , vol.13 , pp. 391-400
    • Schambach, A.1    Bohne, J.2    Chandra, S.3    Will, E.4    Margison, G.P.5    Williams, D.A.6
  • 44
    • 0031710033 scopus 로고    scopus 로고
    • A third-generation lentivirus vector with a conditional packaging system
    • Dull T, Zufferey R, Kelly M, Mandel RJ, Nguyen M, Trono D et al. A third-generation lentivirus vector with a conditional packaging system. J Virol 1998; 72: 8463-8471.
    • (1998) J Virol , vol.72 , pp. 8463-8471
    • Dull, T.1    Zufferey, R.2    Kelly, M.3    Mandel, R.J.4    Nguyen, M.5    Trono, D.6
  • 45
    • 60249101799 scopus 로고    scopus 로고
    • Wnt3a deficiency irreversibly impairs hematopoietic stem cell self-renewal and leads to defects in progenitor cell differentiation
    • Luis TC, Weerkamp F, Naber BA, Baert MR, de Haas EF, Nikolic T et al. Wnt3a deficiency irreversibly impairs hematopoietic stem cell self-renewal and leads to defects in progenitor cell differentiation. Blood 2009; 113: 546-554.
    • (2009) Blood , vol.113 , pp. 546-554
    • Luis, T.C.1    Weerkamp, F.2    Naber, B.A.3    Baert, M.R.4    De Haas, E.F.5    Nikolic, T.6
  • 47
    • 48749101937 scopus 로고    scopus 로고
    • WNT signalling in the immune system: WNT is spreading its wings
    • Staal FJ, Luis TC, Tiemessen MM. WNT signalling in the immune system: WNT is spreading its wings. Nat Rev Immunol 2008; 8: 581-593.
    • (2008) Nat Rev Immunol , vol.8 , pp. 581-593
    • Staal, F.J.1    Luis, T.C.2    Tiemessen, M.M.3
  • 48
    • 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, Rudolph C, Meyer J, Li Z 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    Rudolph, C.4    Meyer, J.5    Li, Z.6
  • 49
    • 0033571370 scopus 로고    scopus 로고
    • Correction of X-linked immunodeficient mice by competitive reconstitution with limiting numbers of normal bone marrow cells
    • Rohrer J, Conley ME. Correction of X-linked immunodeficient mice by competitive reconstitution with limiting numbers of normal bone marrow cells. Blood 1999; 94: 3358-3365.
    • (1999) Blood , vol.94 , pp. 3358-3365
    • Rohrer, J.1    Conley, M.E.2
  • 50
    • 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. Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells. Gene Ther 2009; 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
  • 51
    • 25144488563 scopus 로고    scopus 로고
    • HIV-mediated expression of Btk in hematopoietic stem cells is not sufficient to restore B cell function in X-linked immunodeficient mice
    • Tanabe H, Miyake K, Shimada T. HIV-mediated expression of Btk in hematopoietic stem cells is not sufficient to restore B cell function in X-linked immunodeficient mice. J Nippon Med Sch 2005; 72: 203-212.
    • (2005) J Nippon Med Sch , vol.72 , pp. 203-212
    • Tanabe, H.1    Miyake, K.2    Shimada, T.3
  • 52
    • 33947210222 scopus 로고    scopus 로고
    • Impaired Toll-like receptor 8-mediated IL-6 and TNF-alpha production in antigen-presenting cells from patients with X-linked agammaglobulinemia
    • Sochorova K, Horvath R, Rozkova D, Litzman J, Bartunkova J, Sediva A et al. Impaired Toll-like receptor 8-mediated IL-6 and TNF-alpha production in antigen-presenting cells from patients with X-linked agammaglobulinemia. Blood 2007; 109: 2553-2556.
    • (2007) Blood , vol.109 , pp. 2553-2556
    • Sochorova, K.1    Horvath, R.2    Rozkova, D.3    Litzman, J.4    Bartunkova, J.5    Sediva, A.6
  • 53
    • 0037815206 scopus 로고    scopus 로고
    • Brutons tyrosine kinase cooperates with the B cell linker protein SLP-65 as a tumor suppressor in pre-B cells
    • Kersseboom R, Middendorp S, Dingjan GM, Dahlenborg K, Reth M, Jumaa H et al. Brutons tyrosine kinase cooperates with the B cell linker protein SLP-65 as a tumor suppressor in pre-B cells. J Exp Med 2003; 198: 91-98.
    • (2003) J Exp Med , vol.198 , pp. 91-98
    • Kersseboom, R.1    Middendorp, S.2    Dingjan, G.M.3    Dahlenborg, K.4    Reth, M.5    Jumaa, H.6
  • 54
    • 0030758466 scopus 로고    scopus 로고
    • The X-linked immunodeficiency defect in the mouse is corrected by expression of human Brutons tyrosine kinase from a yeast artificial chromosome transgene
    • Maas A, Dingjan GM, Savelkoul HF, Kinnon C, Grosveld F, Hendriks RW. The X-linked immunodeficiency defect in the mouse is corrected by expression of human Brutons tyrosine kinase from a yeast artificial chromosome transgene. Eur J Immunol 1997; 27: 2180-2187.
    • (1997) Eur J Immunol , vol.27 , pp. 2180-2187
    • Maas, A.1    Dingjan, G.M.2    Savelkoul, H.F.3    Kinnon, C.4    Grosveld, F.5    Hendriks, R.W.6
  • 55
    • 54049090008 scopus 로고    scopus 로고
    • Sola dosis facit venenum Leukemia in gene therapy trials: A question of vectors, inserts and dosage?
    • Staal FJ, Pike-Overzet K, Ng YY, van Dongen JJ. Sola dosis facit venenum. Leukemia in gene therapy trials: a question of vectors, inserts and dosage? Leukemia 2008; 22: 1849-1852.
    • (2008) Leukemia , vol.22 , pp. 1849-1852
    • Staal, F.J.1    Pike-Overzet, K.2    Ng, Y.Y.3    Van Dongen, J.J.4
  • 57
    • 44849140430 scopus 로고    scopus 로고
    • Gene therapy of inherited diseases
    • Fischer A, Cavazzana-Calvo M. Gene therapy of inherited diseases. Lancet 2008 371 2044-2047.
    • (2008) Lancet , vol.371 , pp. 2044-2047
    • Fischer, A.1    Cavazzana-Calvo, M.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.