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Preclinical models of mastocytosis
80. Evans E, Gardino A, Hodous B, et al. Blu-285, a potent and selective inhibitor for hemato- logic malignancies with KIT exon 17 muta- tions. Blood. 2015;126(23):568-568.
81. Evans EK, Gardino AK, Kim JL, et al. A pre- cision therapy against cancers driven by KIT/PDGFRA mutations. Sci Tran Med. 2017;9(414).
82. Lim KH, Tefferi A, Lasho TL, et al. Systemic mastocytosis in 342 consecutive adults: sur- vival studies and prognostic factors. Blood. 2009;113(23):5727-5736.
83. Chaix A, Lopez S, Voisset E, Gros L, Dubreuil P, De Sepulveda P. Mechanisms of STAT protein activation by oncogenic KIT mutants in neoplastic mast cells. J Biol Chem. 2011;286(8):5956-5966.
84. Page BD, Khoury H, Laister RC, et al. Small molecule STAT5-SH2 domain inhibitors exhibit potent antileukemia activity. J Med Chem. 2012;55(3):1047-1055.
85. Peter B, Bibi S, Eisenwort G, et al. Drug- induced inhibition of phosphorylation of STAT5 overrides drug resistance in neoplastic mast cells. Leukemia. 2018;32(4):1016-1022.
86. Gabillot-Carre M, Lepelletier Y, Humbert M, et al. Rapamycin inhibits growth and sur- vival of D816V-mutated c-kit mast cells. Blood. 2006;108(3):1065-1072.
87. Blatt K, Herrmann H, Mirkina I, et al. The PI3-kinase/mTOR-targeting drug NVP- BEZ235 inhibits growth and IgE-dependent activation of human mast cells and basophils. PLoS One. 2012;7(1):e29925.
88. Parikh SA, Kantarjian HM, Richie MA, Cortes JE, Verstovsek S. Experience with everolimus (RAD001), an oral mammalian target of rapamycin inhibitor, in patients with systemic mastocytosis. Leuk Lymphoma. 2010;51(2):269-274.
89. Baldus SE, Zirbes TK, Thiele J, Eming SA, Henz BM, Hartmann K. Altered apoptosis and cell cycling of mast cells in bone marrow lesions of patients with systemic mastocyto- sis. Haematologica. 2004;89(12):1525-1527.
90. Cervero C, Escribano L, San Miguel JF, et al. Expression of Bcl-2 by human bone marrow mast cells and its overexpression in mast cell leukemia. Am J Hematol. 1999;60(3):191- 195.
91. Hartmann K, Artuc M, Baldus SE, et al. Expression of Bcl-2 and Bcl-xL in cutaneous and bone marrow lesions of mastocytosis. Am J Pathol. 2003;163(3):819-826.
92. Aichberger KJ, Mayerhofer M, Gleixner KV, et al. Identification of MCL1 as a novel tar- get in neoplastic mast cells in systemic mas- tocytosis: inhibition of mast cell survival by MCL1 antisense oligonucleotides and syner- gism with PKC412. Blood. 2007;109(7): 3031-3041.
93. Aichberger KJ, Gleixner KV, Mirkina I, et al. Identification of proapoptotic Bim as a tumor suppressor in neoplastic mast cells: role of KIT D816V and effects of various tar- geted drugs. Blood. 2009;114(26):5342-5351.
94. Peter B, Cerny-Reiterer S, Hadzijusufovic E, et al. The pan-Bcl-2 blocker obatoclax pro- motes the expression of Puma, Noxa, and Bim mRNA and induces apoptosis in neo- plastic mast cells. J Leukoc Biol. 2014;95(1): 95-104.
95. Valent P, Cerny-Reiterer S, Herrmann H, et al. Phenotypic heterogeneity, novel diagnos- tic markers, and target expression profiles in normal and neoplastic human mast cells. Best Pract Res Clin Haematol. 2010;23(3): 369-378.
96. Teodosio C, Mayado A, Sanchez-Munoz L, et al. The immunophenotype of mast cells and its utility in the diagnostic work-up of systemic mastocytosis. J Leukoc Biol. 2015;97(1):49-59.
97. Pardanani A, Lasho T, Chen D, et al. Aberrant expression of CD123 (interleukin- 3 receptor alpha) on neoplastic mast cells. Leukemia. 2015;29(7):1605-1608.
98. Sotlar K, Cerny-Reiterer S, Petat-Dutter K, et al. Aberrant expression of CD30 in neoplas- tic mast cells in high-grade mastocytosis. Mod Pathol. 2011;24(4):585-595.
99. Blatt K, Cerny-Reiterer S, Schwaab J, et al. Identification of the Ki-1 antigen (CD30) as a novel therapeutic target in systemic masto- cytosis. Blood. 2015;126(26):2832-2841.
100. Florian S, Sonneck K, Hauswirth AW, et al. Detection of molecular targets on the sur- face of CD34+/CD38-- stem cells in various myeloid malignancies. Leuk Lymphoma. 2006;47(2):207-222.
101. Krauth MT, Bohm A, Agis H, et al. Effects of the CD33-targeted drug gemtuzumab ozogamicin (Mylotarg) on growth and mediator secretion in human mast cells and blood basophils. Exp Hematol. 2007;35(1): 108-116.
102. Alvarez-Twose I, Martinez-Barranco P, Gotlib J, et al. Complete response to gem-
tuzumab ozogamicin in a patient with refractory mast cell leukemia. Leukemia. 2016;30(8):1753-1756.
103. Zuber J, Shi J, Wang E, et al. RNAi screen identifies Brd4 as a therapeutic target in acute myeloid leukaemia. Nature. 2011;478(7370):524-528.
104. Herrmann H, Blatt K, Shi J, et al. Small-mol- ecule inhibition of BRD4 as a new potent approach to eliminate leukemic stem- and progenitor cells in acute myeloid leukemia AML. Oncotarget. 2012;3(12):1588-1599.
105.Filippakopoulos P, Qi J, Picaud S, et al. Selective inhibition of BET bromodomains. Nature. 2010;468(7327):1067-1073.
106. Chaidos A, Caputo V, Gouvedenou K, et al. Potent antimyeloma activity of the novel bromodomain inhibitors I-BET151 and I- BET762. Blood. 2014;123(5):697-705.
107. Wyspianska BS, Bannister AJ, Barbieri I, et al. BET protein inhibition shows efficacy against JAK2V617F-driven neoplasms. Leukemia. 2014;28(1):88-97.
108. Mirguet O, Gosmini R, Toum J, et al. Discovery of epigenetic regulator I-BET762: lead optimization to afford a clinical candi- date inhibitor of the BET bromodomains. J Med Chem. 2013;56(19):7501-7515.
109. Wedeh G, Cerny-Reiterer S, Eisenwort G, et al. Identification of bromodomain-contain- ing protein-4 as a novel marker and epige- netic target in mast cell leukemia. Leukemia. 2015;29(11):2230-2237.
110. Zappulla JP, Dubreuil P, Desbois S, et al. Mastocytosis in mice expressing human Kit receptor with the activating Asp816Val mutation. J Exp Med. 2005;202(12):1635- 1641.
111. Gerbaulet A, Wickenhauser C, Scholten J, et al. Mast cell hyperplasia, B-cell malignancy, and intestinal inflammation in mice with conditional expression of a constitutively active Kit. Blood. 2011;117(6):2012-2021.
112. Schumacher U, van Damme EJ, Peumans WJ, Butterfield JH, Mitchell BS. Lectin histo- chemistry of human leukaemic mast cells (HMC-1) transplanted into severe combined immunodeficient (scid) mice. Acta Histochem. 1998;100(1):1-9.
113. Bibi S, Zhang Y, Hugonin C, et al. A new humanized in vivo model of KIT D816V+ advanced systemic mastocytosis monitored using a secreted luciferase. Oncotarget. 2016;7(50):82985-83000.
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