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Volumn 27, Issue 10, 2016, Pages 847-859

High Level of Perforin Expression is Required for Effective Correction of Hemophagocytic Lymphohistiocytosis

Author keywords

[No Author keywords available]

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; LENTIVIRUS VECTOR; MICRORNA 126; PERFORIN; PHOSPHOGLYCERATE KINASE; MICRORNA;

EID: 84989271383     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2016.065     Document Type: Article
Times cited : (10)

References (38)
  • 1
    • 33845619137 scopus 로고    scopus 로고
    • HLH-2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis
    • Henter JI, Horne A, Arico M et al. HLH-2004: diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer 2007;48:124-131.
    • (2007) Pediatr Blood Cancer , vol.48 , pp. 124-131
    • Henter, J.I.1    Horne, A.2    Arico, M.3
  • 2
    • 84856108251 scopus 로고    scopus 로고
    • Hemophagocytic lymphohistiocytosis: Updates and evolving concepts
    • Risma K, Jordan MB. Hemophagocytic lymphohistiocytosis: updates and evolving concepts. Curr Opin Pediatr 2012;24:9-15.
    • (2012) Curr Opin Pediatr , vol.24 , pp. 9-15
    • Risma, K.1    Jordan, M.B.2
  • 3
    • 84878384497 scopus 로고    scopus 로고
    • Hemophagocytic lymphohistiocytosis: An update on diagnosis and pathogenesis
    • Rosado FG, Kim AS. Hemophagocytic lymphohistiocytosis: An update on diagnosis and pathogenesis. Am J Clin Pathol 2013;139:713-727.
    • (2013) Am J Clin Pathol , vol.139 , pp. 713-727
    • Rosado, F.G.1    Kim, A.S.2
  • 4
    • 80054109736 scopus 로고    scopus 로고
    • How i treat hemophagocytic lymphohistiocytosis
    • Jordan MB, Allen CE, Weitzman S, et al. How I treat hemophagocytic lymphohistiocytosis. Blood 2011;118:4041-4052.
    • (2011) Blood , vol.118 , pp. 4041-4052
    • Jordan, M.B.1    Allen, C.E.2    Weitzman, S.3
  • 5
    • 82155184541 scopus 로고    scopus 로고
    • Hypomorphic mutations in PRF1, MUNC13-4, and STXBP2 are associated with adult-onset familial HLH
    • Zhang K, Jordan MB, Marsh RA, et al. Hypomorphic mutations in PRF1, MUNC13-4, and STXBP2 are associated with adult-onset familial HLH. Blood 2011;118:5794-5798.
    • (2011) Blood , vol.118 , pp. 5794-5798
    • Zhang, K.1    Jordan, M.B.2    Marsh, R.A.3
  • 6
    • 79551703613 scopus 로고    scopus 로고
    • Familial haemophagocytic lymphohistiocytosis: Advances in the genetic basis, diagnosis and management
    • Gholam C, Grigoriadou S, Gilmour KC, et al. Familial haemophagocytic lymphohistiocytosis: Advances in the genetic basis, diagnosis and management. Clin Exp Immunol 2011;163:271-283.
    • (2011) Clin Exp Immunol , vol.163 , pp. 271-283
    • Gholam, C.1    Grigoriadou, S.2    Gilmour, K.C.3
  • 7
    • 0033520970 scopus 로고    scopus 로고
    • Perforin gene defects in familial hemophagocytic lymphohistiocytosis
    • Stepp SE, Dufourcq-Lagelouse R, Le Deist F, et al. Perforin gene defects in familial hemophagocytic lymphohistiocytosis. Science 1999; 286:1957-1959.
    • (1999) Science , vol.286 , pp. 1957-1959
    • Stepp, S.E.1    Dufourcq-Lagelouse, R.2    Le Deist, F.3
  • 8
    • 33845199637 scopus 로고    scopus 로고
    • Perforinmediated target-cell death and immune homeostasis
    • Voskoboinik I, Smyth MJ, Trapani JA. Perforinmediated target-cell death and immune homeostasis. Nat Rev Immunol 2006;6:940-952.
    • (2006) Nat Rev Immunol , vol.6 , pp. 940-952
    • Voskoboinik, I.1    Smyth, M.J.2    Trapani, J.A.3
  • 9
    • 84876174174 scopus 로고    scopus 로고
    • Perforin forms transient pores on the target cell plasma membrane to facilitate rapid access of granzymes during killer cell attack
    • Lopez JA, Susanto O, Jenkins MR, et al. Perforin forms transient pores on the target cell plasma membrane to facilitate rapid access of granzymes during killer cell attack. Blood 2013;121:2659-2668.
    • (2013) Blood , vol.121 , pp. 2659-2668
    • Lopez, J.A.1    Susanto, O.2    Jenkins, M.R.3
  • 10
    • 15944399347 scopus 로고    scopus 로고
    • Perforin and lymphohistiocytic proliferative disorders
    • Katano H, Cohen JI. Perforin and lymphohistiocytic proliferative disorders. Br J Haematol 2005; 128:739-750.
    • (2005) Br J Haematol , vol.128 , pp. 739-750
    • Katano, H.1    Cohen, J.I.2
  • 11
    • 84885730018 scopus 로고    scopus 로고
    • An intermediate alemtuzumab schedule reduces the incidence of mixed chimerism following reduced-intensity conditioning hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis
    • Marsh RA, Kim MO, Liu C, et al. An intermediate alemtuzumab schedule reduces the incidence of mixed chimerism following reduced-intensity conditioning hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis. Biol Blood Marrow Transplant 2013;19:1625-1631.
    • (2013) Biol Blood Marrow Transplant , vol.19 , pp. 1625-1631
    • Marsh, R.A.1    Kim, M.O.2    Liu, C.3
  • 12
    • 84886727845 scopus 로고    scopus 로고
    • Hemophagocytic lymphohistiocytosis: Advances in pathophysiology, diagnosis, and treatment
    • Chandrakasan S, Filipovich AH. Hemophagocytic lymphohistiocytosis: Advances in pathophysiology, diagnosis, and treatment. J Pediatr 2013;163: 1253-1259.
    • (2013) J Pediatr , vol.163 , pp. 1253-1259
    • Chandrakasan, S.1    Filipovich, A.H.2
  • 13
    • 84891505988 scopus 로고    scopus 로고
    • Mixed hematopoietic or T-cell chimerism above a minimal threshold restores perforin-dependent immune regulation in perforindeficient mice
    • Terrell CE, Jordan MB. Mixed hematopoietic or T-cell chimerism above a minimal threshold restores perforin-dependent immune regulation in perforindeficient mice. Blood 2013;122:2618-2621.
    • (2013) Blood , vol.122 , pp. 2618-2621
    • Terrell, C.E.1    Jordan, M.B.2
  • 14
    • 3242752040 scopus 로고    scopus 로고
    • An animal model of hemophagocytic lymphohistiocytosis (HLH): CD8+ T cells and interferon gamma are essential for the disorder
    • Jordan MB, Hildeman D, Kappler J, et al. An animal model of hemophagocytic lymphohistiocytosis (HLH): CD8+ T cells and interferon gamma are essential for the disorder. Blood 2004;104: 735-743.
    • (2004) Blood , vol.104 , pp. 735-743
    • Jordan, M.B.1    Hildeman, D.2    Kappler, J.3
  • 15
    • 84926979642 scopus 로고    scopus 로고
    • Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency
    • Carmo M, Risma KA, Arumugam P, et al. Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency. Mol Ther 2015;23:737-745.
    • (2015) Mol Ther , vol.23 , pp. 737-745
    • Carmo, M.1    Risma, K.A.2    Arumugam, P.3
  • 16
    • 79958231379 scopus 로고    scopus 로고
    • Gene therapy for canine leukocyte adhesion deficiency with lentiviral vectors using the murine stem cell virus and human phosphoglycerate kinase promoters
    • Hunter MJ, Zhao H, Tuschong LM, et al. Gene therapy for canine leukocyte adhesion deficiency with lentiviral vectors using the murine stem cell virus and human phosphoglycerate kinase promoters. Hum Gene Ther 2011;22:689-696.
    • (2011) Hum Gene Ther , vol.22 , pp. 689-696
    • Hunter, M.J.1    Zhao, H.2    Tuschong, L.M.3
  • 17
    • 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, et al. Lineage-and stage-restricted lentiviral vectors for the gene therapy of chronic granulomatous disease. Gene Ther 2011;18:1087-1097.
    • (2011) Gene Ther , vol.18 , pp. 1087-1097
    • Barde, I.1    Laurenti, E.2    Verp, S.3
  • 18
    • 84905451458 scopus 로고    scopus 로고
    • Dualregulated lentiviral vector for gene therapy of Xlinked chronic granulomatosis
    • Chiriaco M, Farinelli G, Capo V, et al. Dualregulated lentiviral vector for gene therapy of Xlinked chronic granulomatosis. Mol Ther 2014; 22:1472-1483.
    • (2014) Mol Ther , vol.22 , pp. 1472-1483
    • Chiriaco, M.1    Farinelli, G.2    Capo, V.3
  • 19
    • 84879867061 scopus 로고    scopus 로고
    • Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
    • Aiuti A, Biasco L, Scaramuzza S, et al. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science 2013; 341:1233151.
    • (2013) Science , vol.341 , pp. 1233151
    • Aiuti, A.1    Biasco, L.2    Scaramuzza, S.3
  • 20
    • 84928389399 scopus 로고    scopus 로고
    • Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome
    • Hacein-Bey Abina S, Gaspar HB, Blondeau J, et al. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. JAMA 2015;313: 1550-1563.
    • (2015) JAMA , vol.313 , pp. 1550-1563
    • Hacein-Bey Abina, S.1    Gaspar, H.B.2    Blondeau, J.3
  • 21
    • 84891071036 scopus 로고    scopus 로고
    • Etoposide selectively ablates activated T cells to control the immunoregulatory disorder hemophagocytic lymphohistiocytosis
    • Johnson TS, Terrell CE, Millen SH, et al. Etoposide selectively ablates activated T cells to control the immunoregulatory disorder hemophagocytic lymphohistiocytosis. J Immunol 2014; 192:84-91.
    • (2014) J Immunol , vol.192 , pp. 84-91
    • Johnson, T.S.1    Terrell, C.E.2    Millen, S.H.3
  • 22
    • 79959356948 scopus 로고    scopus 로고
    • Protection from endogenous perforin: Glycans and the C terminus regulate exocytic trafficking in cytotoxic lymphocytes
    • Brennan AJ, Chia J, Browne KA, et al. Protection from endogenous perforin: glycans and the C terminus regulate exocytic trafficking in cytotoxic lymphocytes. Immunity 2011;34:879-892.
    • (2011) Immunity , vol.34 , pp. 879-892
    • Brennan, A.J.1    Chia, J.2    Browne, K.A.3
  • 23
    • 0031867025 scopus 로고    scopus 로고
    • NK cells differentiated from bone marrow, cord blood and peripheral blood stem cells exhibit similar phenotype and functions
    • Carayol G, Robin C, Bourhis JH, et al. NK cells differentiated from bone marrow, cord blood and peripheral blood stem cells exhibit similar phenotype and functions. Eur J Immunol 1998;28: 1991-2002.
    • (1998) Eur J Immunol , vol.28 , pp. 1991-2002
    • Carayol, G.1    Robin, C.2    Bourhis, J.H.3
  • 24
    • 0030250754 scopus 로고    scopus 로고
    • Vaccination protects b2 microglobulin deficient mice from immune mediated mortality but not from persisting viral infection
    • Hildeman D, Salvato M, Whitton JL, et al. Vaccination protects b2 microglobulin deficient mice from immune mediated mortality but not from persisting viral infection. Vaccine 1996;14:1223-1229.
    • (1996) Vaccine , vol.14 , pp. 1223-1229
    • Hildeman, D.1    Salvato, M.2    Whitton, J.L.3
  • 25
    • 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, et al. Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med 2010;2:58ra84.
    • (2010) Sci Transl Med , vol.2 , pp. 58ra84
    • Gentner, B.1    Visigalli, I.2    Hiramatsu, H.3
  • 26
    • 77957252821 scopus 로고    scopus 로고
    • Comprehensive microRNA expression profiling of the hematopoietic hierarchy
    • Petriv OI, Kuchenbauer F, Delaney AD, et al. Comprehensive microRNA expression profiling of the hematopoietic hierarchy. Proc Natl Acad Sci U S A 2010;107:15443-15448.
    • (2010) Proc Natl Acad Sci U S A , vol.107 , pp. 15443-15448
    • Petriv, O.I.1    Kuchenbauer, F.2    Delaney, A.D.3
  • 27
    • 78649242699 scopus 로고    scopus 로고
    • Nextgeneration sequencing identifies the natural killer cell microRNA transcriptome
    • Fehniger TA, Wylie T, Germino E, et al. Nextgeneration sequencing identifies the natural killer cell microRNA transcriptome. Genome Res 2010; 20:1590-1604.
    • (2010) Genome Res , vol.20 , pp. 1590-1604
    • Fehniger, T.A.1    Wylie, T.2    Germino, E.3
  • 28
    • 34250877841 scopus 로고    scopus 로고
    • A mammalian microRNA expression atlas based on small RNA library sequencing
    • Landgraf P, Rusu M, Sheridan R, et al. A mammalian microRNA expression atlas based on small RNA library sequencing. Cell 2007;129:1401-1414.
    • (2007) Cell , vol.129 , pp. 1401-1414
    • Landgraf, P.1    Rusu, M.2    Sheridan, R.3
  • 29
    • 0028815676 scopus 로고
    • Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells
    • Challita PM, Skelton D, el-Khoueiry A, et al. Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells. J Virol 1995; 69:748-755.
    • (1995) J Virol , vol.69 , pp. 748-755
    • Challita, P.M.1    Skelton, D.2    El-Khoueiry, A.3
  • 30
    • 0030862341 scopus 로고    scopus 로고
    • Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells
    • Robbins PB, Yu XJ, Skelton DM, et al. Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells. J Virol 1997;71:9466-9474.
    • (1997) J Virol , vol.71 , pp. 9466-9474
    • Robbins, P.B.1    Yu, X.J.2    Skelton, D.M.3
  • 31
    • 84940021026 scopus 로고    scopus 로고
    • Live cell evaluation of granzyme delivery and death receptor signaling in tumor cells targeted by human natural killer cells
    • Vrazo AC, Hontz AE, Figueira SK, et al. Live cell evaluation of granzyme delivery and death receptor signaling in tumor cells targeted by human natural killer cells. Blood 2015;126:e1-e10.
    • (2015) Blood , vol.126 , pp. e1-e10
    • Vrazo, A.C.1    Hontz, A.E.2    Figueira, S.K.3
  • 32
    • 77951699510 scopus 로고    scopus 로고
    • The transcriptional control of the perforin locus
    • Pipkin ME, Rao A, Lichtenheld MG. The transcriptional control of the perforin locus. Immunol Rev 2010;235:55-72.
    • (2010) Immunol Rev , vol.235 , pp. 55-72
    • Pipkin, M.E.1    Rao, A.2    Lichtenheld, M.G.3
  • 33
    • 84886662672 scopus 로고    scopus 로고
    • Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors
    • Koldej RM, Carney G, Wielgosz MM, et al. Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors. Hum Gene Ther Clin Dev 2013;24: 77-85.
    • (2013) Hum Gene Ther Clin Dev , vol.24 , pp. 77-85
    • Koldej, R.M.1    Carney, G.2    Wielgosz, M.M.3
  • 34
    • 84857579405 scopus 로고    scopus 로고
    • Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy
    • Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al. Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy. Methods Enzymol 2012;507:187-198.
    • (2012) Methods Enzymol , vol.507 , pp. 187-198
    • Cartier, N.1    Hacein-Bey-Abina, S.2    Bartholomae, C.C.3
  • 35
    • 70449427834 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy
    • Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009;326:818-823.
    • (2009) Science , vol.326 , pp. 818-823
    • Cartier, N.1    Hacein-Bey-Abina, S.2    Bartholomae, C.C.3
  • 36
    • 84870894496 scopus 로고    scopus 로고
    • Attenuation of miR-126 activity expands HSC in vivo without exhaustion
    • Lechman ER, Gentner B, van Galen P, et al. Attenuation of miR-126 activity expands HSC in vivo without exhaustion. Cell Stem Cell 2012;11:799-811.
    • (2012) Cell Stem Cell , vol.11 , pp. 799-811
    • Lechman, E.R.1    Gentner, B.2    Van Galen, P.3
  • 37
    • 84884187508 scopus 로고    scopus 로고
    • Perforin deficiency impairs a critical immunoregulatory loop involving murine CD8+ T cells and dendritic cells
    • Terrell CE, Jordan MB. Perforin deficiency impairs a critical immunoregulatory loop involving murine CD8+ T cells and dendritic cells. Blood 2013;121:5184-5191.
    • (2013) Blood , vol.121 , pp. 5184-5191
    • Terrell, C.E.1    Jordan, M.B.2
  • 38
    • 79960714121 scopus 로고    scopus 로고
    • Perforin is a critical physiologic regulator of T-cell activation
    • Lykens JE, Terrell CE, Zoller EE, et al. Perforin is a critical physiologic regulator of T-cell activation. Blood 2011;118:618-626.
    • (2011) Blood , vol.118 , pp. 618-626
    • Lykens, J.E.1    Terrell, C.E.2    Zoller, E.E.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.