Targeting Notch signaling in autoimmune and lymphoproliferative disease

Blood

Volumn 111, Issue 2, 2008, Pages 705-714

  Teachey, David T ^a,c   Seif, Alix E ^a   Brown, Valerie I ^a   Bruno, Marlo ^a   Bunte, Ralph M ^a   Chang, Yueh J ^a   Choi, John K ^a   Fish, Jonathan D ^a   Hall, Junior ^a   Reid, Gregor S ^a   Ryan, Theresa ^a   Sheen, Cecilia ^a   Zweidler McKay, Patrick ^b   Grupp, Stephan A ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

^c CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DOUBLE STRANDED DNA; GAMMA SECRETASE INHIBITOR; N S PHENYLGLYCINE TERT BUTYLESTER; NOTCH RECEPTOR; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; AUTOIMMUNE DISEASE; CONTROLLED STUDY; CYTOKINE PRODUCTION; ENZYME LINKED IMMUNOSORBENT ASSAY; LYMPHOID TISSUE; LYMPHOPROLIFERATIVE DISEASE; MOUSE; NEPHRITIS; NONHUMAN; PRIORITY JOURNAL; RANDOMIZATION; SIGNAL TRANSDUCTION; SYSTEMIC LUPUS ERYTHEMATOSUS; T LYMPHOCYTE ACTIVATION;

EID: 38349095890     PISSN: 00064971     EISSN: 00064971     Source Type: Journal    
DOI: 10.1182/blood-2007-05-087353     Document Type: Article

References (60)

1. Bleesing JJ, Brown MR, Novicio C, et al. A composite picture of TcR alpha/beta(+) CD4(-)CD8(-) T Cells (alpha/beta-DNTCs) in humans with autoimmune lymphoproliferative syndrome. Clin Immunol. 2002;104:21-30.
2. Shi X, Xie C, Kreska D, Richardson JA, Mohan C. Genetic dissection of SLE: SLE1 and FAS impact alternate pathways leading to lymphoproliferative autoimmunity. J Exp Med. 2002;196:281-292.
3. Dean GS, Anand A, Blofeld A, Isenberg DA, Lydyard PM. Characterization of CD3+ CD4- CD8-(double negative) T cells in patients with systemic lupus erythematosus: production of IL-4. Lupus. 2002;11:501-507.
4. Xue C, Lan-Lan W, Bei C, Jie C, Wei-Hua F. Abnormal Fas/FasL and caspase-3-mediated apoptotic signaling pathways of T lymphocyte subset in patients with systemic lupus erythematosus. Cell Immunol. 2006;239:121-128.
5. Teachey DT, Manno CS, Axsom KM, et al. Unmasking Evans syndrome: T-cell phenotype and apoptotic response reveal autoimmune lymphoproliferative syndrome (ALPS). Blood. 2005;105:2443-2448.
6. Watanabe-Fukunaga R, Brannan CI, Copeland NG, Jenkins NA, Nagata S. Lymphoproliferation disorder in mice explained by defects in Fas antigen that mediates apoptosis. Nature. 1992;356:314-317.
7. Cohen PL, Eisenberg RA. Lpr and gld: single gene models of systemic autoimmunity and lymphoproliferative disease. Annu Rev Immunol. 1991;9:243-269.
8. Bleesing JJ, Straus SE, Fleisher TA. Autoimmune lymphoproliferative syndrome. A human disorder of abnormal lymphocyte survival. Pediatr Clin North Am. 2000;47:1291-1310.
9. Straus SE, Sneller M, Lenardo MJ, Puck JM, Strober W. An inherited disorder of lymphocyte apoptosis: the autoimmune lymphoproliferative syndrome. Ann Intern Med. 1999;130:591-601.
10. Sneller MC, Straus SE, Jaffe ES, et al. A novel lymphoproliferative/ autoimmune syndrome resembling murine lpr/gld disease. J Clin Invest. 1992;90:334-341.
11. Rieux-Laucat F, Le Deist F, Fischer A. Autoimmune lymphoproliferative syndromes: genetic defects of apoptosis pathways. Cell Death Differ. 2003;10:124-133.
12. Lam GK, Petri M. Assessment of systemic lupus erythematosus. Clin Exp Rheumatol. 2005;23:S120-132.
13. Maillard I, Fang T, Pear WS. Regulation of lymphoid development, differentiation, and function by the Notch pathway. Annu Rev Immunol. 2005;23:945-974.
14. Kimura M, Matsuzawa A. Autoimmunity in mice bearing lprcg: a novel mutant gene. Int Rev Immunol. 1994;11:193-210.
15. Rodriguez W, Mold C, Marnell LL, et al. Prevention and reversal of nephritis in MRL/lpr mice with a single injection of C-reactive protein. Arthritis Rheum. 2006;54:325-335.
16. Sadanaga A, Nakashima H, Masutani K, et al. Amelioration of autoimmune nephritis by imatinib in MRL/lpr mice. Arthritis Rheum. 2005;52:3987-3996.
17. Reilly CM, Mishra N, Miller JM, et al. Modulation of renal disease in MRL/lpr mice by suberoylanilide hydroxamic acid. J Immunol. 2004;173:4171-4178.
18. Furukawa F, Yoshimasu T. Animal models of spontaneous and drug-induced cutaneous lupus erythematosus. Autoimmun Rev. 2005;4:345-350.
19. Churcher I, Beher D. Gamma-secretase as a therapeutic target for the treatment of Alzheimer's disease. Curr Pharm Des. 2005;11:3363-3382.
20. Grabher C, von Boehmer H, Look AT. Notch 1 activation in the molecular pathogenesis of T-cell acute lymphoblastic leukaemia. Nat Rev Cancer. 2006;6:347-359.
21. Hyde LA, McHugh NA, Chen J, et al. Studies to investigate the in vivo therapeutic window of the gamma-secretase inhibitor N2-[(2S)-2-(3,5- difluorophenyl)-2-hydroxyethanoyl]-N1-[(7S)-5-methyl-6-oxo-6, 7-dihydro-5H-dibenzo[b,d]azepin-7-yl]-L-alaninamide (LY411,575) in the CRND8 mouse. J Pharmacol Exp Ther. 2006;319:1133-1143.
22. van Es JH, van Gijn ME, Riccio O, et al. Notch/gamma-secretase inhibition turns proliferative cells in intestinal crypts and adenomas into goblet cells. Nature. 2005;435:959-963.
23. Best JD, Smith DW, Reilly MA, et al. The novel gamma secretase inhibitor N-[cis-4-[(4-chlorophenyl) sulfonyl]-4-(2,5-difluorophenyl)cyclohexyl]-1,1, 1-trifluoromethanesulfonamide (MRK-560) reduces amyloid plaque deposition without evidence of notch-related pathology in the Tg2576 mouse. J Pharmacol Exp Ther. 2007;320:552-558.
24. Wong GT, Manfra D, Poulet FM, et al. Chronic treatment with the gamma-secretase inhibitor LY-411,575 inhibits beta-amyloid peptide production and alters lymphopoiesis and intestinal cell differentiation. J Biol Chem. 2004;279:12876-12882.
25. Comery TA, Martone RL, Aschmies S, et al. Acute gamma-secretase inhibition improves contextual fear conditioning in the Tg2576 mouse model of Alzheimer's disease. J Neurosci. 2005;25:8898-8902.
26. Teachey DT, Obzut DA, Axsom K, et al. Rapamycin improves lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome (ALPS). Blood. 2006;108:1965-1971.
27. Monneaux F, Dumortier H, Steiner G, Briand JP, Muller S. Murine models of systemic lupus erythematosus: B and T cell responses to spliceosomal ribonucleoproteins in MRL/Fas(lpr) and (NZB NZW)F(1) lupus mice. Int Immunol. 2001;13:1155-1163.
28. Watson ML, Rao JK, Gilkeson GS, et al. Genetic analysis of MRL-lpr mice: relationship of the Fas apoptosis gene to disease manifestations and renal disease-modifying loci. J Exp Med. 1992;176:1645-1656.
29. Teachey DT, Obzut DA, Cooperman J, et al. The mTOR inhibitor CCI-779 induces apoptosis and inhibits growth in preclinical models of primary adult human ALL. Blood. 2006;107:1149-1155.
30. Bossu P, Singer GG, Andres P, Ettinger R, Marshak-Rothstein A, Abbas AK. Mature CD4+ T lymphocytes from MRL/lpr mice are resistant to receptor-mediated tolerance and apoptosis. J Immunol. 1993;151:7233-7239.
31. Mosmann T. Rapid colorimetric assay for cellular growth and survival: application to proliferation and cytotoxicity assays. J Immunol Methods. 1983;65:55-63.
32. Dianzani U, Bragardo M, DiFranco D, et al. Deficiency of the Fas apoptosis pathway without Fas gene mutations in pediatric patients with autoimmunity/lymphoproliferation. Blood. 1997;89:2871-2879.
33. Ogata Y, Kimura M, Shimada K, et al. Distinctive expression of lprcg in the heterozygous state on different genetic backgrounds. Cell Immunol. 1993;148:91-102.
34. Bobé P, Bonardelle D, Benihoud K, Opolon P, Chelbi-Alix MK. Arsenic trioxide: A promising novel therapeutic agent for lymphoproliferative and autoimmune syndromes in MRL/lpr mice. Blood. 2006;108:3967-3975.
35. Wolfer A, Wilson A, Nemir M, MacDonald HR, Radtke F. Inactivation of Notch1 impairs VDJbeta rearrangement and allows pre-TCR-independent survival of early alpha beta Lineage Thymocytes. Immunity. 2002;16:869-879.
36. Maillard I, Tu L, Sambandam A, et al. The requirement for Notch signaling at the beta-selection checkpoint in vivo is absolute and independent of the pre-T cell receptor. J Exp Med. 2006;203:2239-2245.
37. Adler SH, Chiffoleau E, Xu L, et al. Notch signaling augments T cell responsiveness by enhancing CD25 expression. J Immunol. 2003;171:2896-2903.
38. Weng AP, Ferrando AA, Lee W, et al. Activating mutations of NOTCH1 in human T cell acute lymphoblastic leukemia. Science. 2004;306:269-271.
39. Shivakumar S, Tsokos GC, Datta SK. T cell receptor alpha/beta expressing double-negative (CD4-/CD8-) and CD4+ T helper cells in humans augment the production of pathogenic anti-DNA autoantibodies associated with lupus nephritis. J Immunol. 1989;143:103-112.
40. Ohga S, Nomura A, Takahata Y, et al. Dominant expression of interleukin 10 but not interferon gamma in CD4(-)CD8(-)alphabetaT cells of autoimmune lymphoproliferative syndrome. Br J Haematol. 2002;119:535-538.
41. Spinozzi F, Agea E, Bistoni O, et al. T lymphocytes bearing the gamma delta T cell receptor are susceptible to steroid-induced programmed cell death. Scand J Immunol. 1995;41:504-508.
42. Rao VK, Dowdell KC, Dale JK, et al. Pyrimethamine treatment does not ameliorate lymphoproliferation or autoimmune disease in MRL/lpr-/- mice or in patients with autoimmune lymphoproliferative syndrome. Am J Hematol. 2007;82:1049-1055.
43. Fischer K, Voelkl S, Heymann J, et al. Isolation and characterization of human antigen-specific TCR alpha beta+ CD4(-)CD8- double-negative regulatory T cells. Blood. 2005;105:2828-2835.
44. Marlies A, Udo G, Juergen B, Bernd S, Herrmann M, Haas JP. The expanded double negative T cell populations of a patient with ALPS are not clonally related to CD4+ or to CD8+ T cells. Autoimmunity. 2007;40:299-301.
45. Bleesing JJ, Brown MR, Dale JK, et al. TcR-alpha/beta(+) CD4(-)CD8(-) T cells in humans with the autoimmune lymphoproliferative syndrome express a novel CD45 isoform that is analogous to murine B220 and represents a marker of altered O-glycan biosynthesis. Clin Immunol. 2001;100:314-324.
46. Rao VK, Dugan F, Dale JK, et al. Use of mycophenolate mofetil for chronic, refractory immune cytopenias in children with autoimmune lymphoproliferative syndrome. Br J Haematol. 2005;129:534-538.
47. van der Werff Ten Bosch J, Schotte P, Ferster A, et al. Reversion of autoimmune lymphoproliferative syndrome with an antimalarial drug: preliminary results of a clinical cohort study and molecular observations. Br J Haematol. 2002;117:176-188.
48. Cambridge G, Leandro MJ, Teodorescu M, et al. B cell depletion therapy in systemic lupus erythematosus: effect on autoantibody and antimicrobial antibody profiles. Arthritis Rheum. 2006;54:3612-3622.
49. Isenberg DA, Ravirajan CT, Rahman A, Kalsi J. The role of antibodies to DNA in systemic lupus erythematosus-a review and introduction to an international workshop on DNA antibodies held in London, May 1996. Lupus. 1997;6:290-304.
50. Dawson J, Miltz W, Mir AK, Wiessner C. Targeting monocyte chemoattractant protein-1 signalling in disease. Expert Opin Ther Targets. 2003;7:35-48.
51. Tesch GH, Maifert S, Schwarting A, Rollins BJ, Kelley VR. Monocyte chemoattractant protein 1-dependent leukocytic infiltrates are responsible for autoimmune disease in MRL-Fas(lpr) mice. J Exp Med. 1999;190:1813-1824.
52. Shimizu S, Nakashima H, Karube K, Ohshima K, Egashira K. Monocyte chemoattractant protein-1 activates a regional Th1 immunoresponse in nephritis of MRL/lpr mice. Clin Exp Rheumatol. 2005;23:239-242.
53. Hasegawa H, Kohno M, Sasaki M, et al. Antagonist of monocyte chemoattractant protein 1 ameliorates the initiation and progression of lupus nephritis and renal vasculitis in MRL/lpr mice. Arthritis Rheum. 2003;48:2555-2566.
54. Seifert T, Bauer J, Weissert R, Fazekas F, Storch MK. Notch1 and its ligand Jagged1 are present in remyelination in a T-cell- and antibody-mediated model of inflammatory demyelination. Acta Neuropathol (Berl). 2007;113:195-203.
55. Jurynczyk M, Jurewicz A, Bielecki B, Raine CS, Selmaj K. Inhibition of Notch signaling enhances tissue repair in an animal model of multiple sclerosis. J Neuroimmunol. 2005;170:3-10.
56. Poppema S, Maggio E, van den Berg A. Development of lymphoma in Autoimmune Lymphoproliferative Syndrome (ALPS) and its relationship to Fas gene mutations. Leuk Lymphoma. 2004;45:423-431.
57. Berman JN, Look AT. Targeting transcription factors in acute leukemia in children. Curr Drug Targets. 2007;8:727-737.
58. Miele L, Golde T, Osborne B. Notch signaling in cancer. Curr Mol Med. 2006;6:905-918.
59. Maekawa Y, Tsukumo S, Chiba S, et al. Delta1-Notch3 interactions bias the functional differentiation of activated CD4+ T cells. Immunity. 2003;19:549-559.
60. Kared H, Adle-Biassette H, Fois E, et al. Jagged2-expressing hematopoietic progenitors promote regulatory T cell expansion in the periphery through notch signaling. Immunity. 2006;25:823-834.