Downregulation of GATA1 drives impaired hematopoiesis in primary myelofibrosis

Journal of Clinical Investigation

Volumn 127, Issue 4, 2017, Pages 1316-1320

  Gilles, Laure ^a,b,c   Arslan, Ahmet Dirim ^a   Marinaccio, Christian ^a   Wen, Qiang Jeremy ^a   Arya, Priyanka ^a   McNulty, Maureen ^a   Yang, Qiong ^a   Zhao, Jonathan C ^a   Konstantinoff, Katerina ^a   Lasho, Terra ^d   Pardanani, Animesh ^d   Stein, Brady ^a   Plo, Isabelle ^b,c   Sundaravel, Sriram ^e   Wickrema, Amittha ^e   Migliaccio, Annarita ^f,g   Gurbuxani, Sandeep ^e   Vainchenker, William ^b,c   Platanias, Leonidas C ^a,h   Tefferi, Ayalew ^d   Crispino, John D ^a   more..

^a NORTHWESTERN UNIVERSITY   (United States)

^b INSERM   (France)

^c Universite Paris Sud   (France)

^d MAYO CLINIC   (United States)

^e UNIVERSITY OF CHICAGO   (United States)

^f MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^g UNIVERSITY OF BOLOGNA   (Italy)

^h JESSE BROWN VA MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CCAAT ENHANCER BINDING PROTEIN; COMPLEMENTARY DNA; CYCLIN DEPENDENT KINASE INHIBITOR 1A; GROWTH FACTOR; GROWTH FACTOR INDEPENDENT 1; GROWTH FACTOR INDEPENDENT 1B; MESSENGER RNA; MITOGEN ACTIVATED PROTEIN KINASE; PROTEIN BAX; PROTEIN KINASE B; PROTEIN P53; RIBOSOMAL PROTEIN S14; RIBOSOME PROTEIN; RIBOSOME RNA; THROMBOCYTE FACTOR 4; TRANSCRIPTION FACTOR GATA 1; TRANSCRIPTION FACTOR GATA 2; UNCLASSIFIED DRUG; GATA1 PROTEIN, HUMAN; GATA1 PROTEIN, MOUSE; RPS14 PROTEIN, HUMAN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CARCINOGENESIS; CELL DIFFERENTIATION; CELL LINEAGE; CELL MATURATION; CELL PROLIFERATION; CHROMOSOME 5Q; CHROMOSOME DELETION; COMPARATIVE STUDY; CONTROLLED STUDY; DOWN REGULATION; ERYTHROID CELL; GENE EXPRESSION; HUMAN; HUMAN CELL; MEGAKARYOCYTE; MEGAKARYOPOIESIS; MOLECULAR PATHOLOGY; MOUSE; MYELODYSPLASTIC SYNDROME; MYELOID METAPLASIA; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROTEIN FUNCTION; SIGNAL TRANSDUCTION; ANIMAL; BIOSYNTHESIS; CHROMOSOME 5; GENETICS; METABOLISM; PATHOLOGY; THROMBOCYTOPOIESIS;

ANIMALS; CHROMOSOME DELETION; CHROMOSOMES, HUMAN, PAIR 5; DOWN-REGULATION; GATA1 TRANSCRIPTION FACTOR; HUMANS; MEGAKARYOCYTES; MICE; PRIMARY MYELOFIBROSIS; RIBOSOMAL PROTEINS; THROMBOPOIESIS;

EID: 85018710687     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI82905     Document Type: Article

References (23)

1. Thiele J, Kvasnicka HM, Müllauer L, Buxhofer-Ausch V, Gisslinger B, Gisslinger H. Essential thrombocythemia versus early primary myelofibrosis: a multicenter study to validate the WHO classification. Blood. 2011;117(21):5710-5718.
2. Ciurea SO, et al. Pivotal contributions of megakaryocytes to the biology of idiopathic myelofibrosis. Blood. 2007;110(3):986-993.
3. Balduini A, et al. In vitro megakaryocyte differentiation and proplatelet formation in Ph-negative classical myeloproliferative neoplasms: distinct patterns in the different clinical phenotypes. PLoS ONE. 2011;6(6):e21015.
4. Bruns I, et al. Megakaryocytes regulate hematopoietic stem cell quiescence through CXCL4 secretion. Nat Med. 2014;20(11):1315-1320.
5. Zhao M, et al. Megakaryocytes maintain homeostatic quiescence and promote post-injury regeneration of hematopoietic stem cells. Nat Med. 2014;20(11):1321-1326.
6. Villeval JL, et al. High thrombopoietin production by hematopoietic cells induces a fatal myeloproliferative syndrome in mice. Blood. 1997;90(11):4369-4383.
7. Vannucchi AM, et al. Development of myelofibrosis in mice genetically impaired for GATA-1 expression (GATA-1(low) mice). Blood. 2002;100(4):1123-1132.
8. Wen QJ, et al. Targeting megakaryocyticinduced fibrosis in myeloproliferative neoplasms by AURKA inhibition. Nat Med. 2015;21(12):1473-1480.
9. Vannucchi AM, et al. Abnormalities of GATA-1 in megakaryocytes from patients with idiopathic myelofibrosis. Am J Pathol. 2005;167(3):849-858.
10. Vannucchi AM, et al. A pathobiologic pathway linking thrombopoietin, GATA-1, and TGF-beta1 in the development of myelofibrosis. Blood. 2005;105(9):3493-3501.
11. Moignard V, et al. Characterization of transcriptional networks in blood stem and progenitor cells using high-throughput single-cell gene expression analysis. Nat Cell Biol. 2013;15(4):363-372.
12. Lambert MP, Wang Y, Bdeir KH, Nguyen Y, Kowalska MA, Poncz M. Platelet factor 4 regulates megakaryopoiesis through low-density lipoprotein receptor-related protein 1 (LRP1) on megakaryocytes. Blood. 2009;114(11):2290-2298.
13. Desterke C, et al. FLT3-mediated p38-MAPK activation participates in the control of megakaryopoiesis in primary myelofibrosis. Cancer Res. 2011;71(8):2901-2915.
14. Ebert BL, et al. Identification of RPS14 as a 5qsyndrome gene by RNA interference screen. Nature. 2008;451(7176):335-339.
15. Mahmood T, Robinson WA, Hamstra RD, Wallner SF. Macrocytic anemia, thrombocytosis and nonlobulated megakaryocytes: the 5q-syndrome, a distinct entity. Am J Med. 1979;66(6):946-950.
16. Narla A, Ebert BL. Ribosomopathies: human disorders of ribosome dysfunction. Blood. 2010;115(16):3196-3205.
17. Ludwig LS, et al. Altered translation of GATA1 in Diamond-Blackfan anemia. Nat Med. 2014;20(7):748-753.
18. Bibikova E, et al. TNF-mediated inflammation represses GATA1 and activates p38 MAP kinase in RPS19-deficient hematopoietic progenitors. Blood. 2014;124(25):3791-3798.
19. Mercher T, et al. The OTT-MAL fusion oncogene activates RBPJ-mediated transcription and induces acute megakaryoblastic leukemia in a knockin mouse model. J Clin Invest. 2009;119(4):852-864.
20. List A, et al. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med. 2006;355(14):1456-1465.
21. Oliva EN, et al. Biological activity of lenalidomide in myelodysplastic syndromes with del5q: results of gene expression profiling from a multicenter phase II study. Ann Hematol. 2013;92(1):25-32.
22. Mesa RA, et al. Lenalidomide and prednisone for myelofibrosis: Eastern Cooperative Oncology Group (ECOG) phase 2 trial E4903. Blood. 2010;116(22):4436-4438.
23. McCann KL, Baserga SJ. Genetics. Mysterious ribosomopathies. Science. 2013;341(6148):849-850.