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For: Connelly JP, Kwon EM, Gao Y, Trivedi NS, Elkahloun AG, Horwitz MS, Cheng L, Liu PP. Targeted correction of RUNX1 mutation in FPD patient-specific induced pluripotent stem cells rescues megakaryopoietic defects. Blood 2014;124:1926-30. [PMID: 25114263 DOI: 10.1182/blood-2014-01-550525] [Citation(s) in RCA: 58] [Impact Index Per Article: 5.8] [Reference Citation Analysis] [What about the content of this article? (0)] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/14/2022]  Open
Number Cited by Other Article(s)
1
Sahoo SS, Khiami M, Wlodarski MW. Inducible pluripotent stem cell models to study bone marrow failure and MDS predisposition syndromes. Exp Hematol 2025;143:104669. [PMID: 39491640 DOI: 10.1016/j.exphem.2024.104669] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/02/2024] [Revised: 10/24/2024] [Accepted: 10/26/2024] [Indexed: 11/05/2024]
2
Mohammadhosseini M, Enright T, Duvall A, Chitsazan A, Lin HY, Ors A, Davis BA, Nikolova O, Bresciani E, Diemer J, Craft K, Menezes AC, Merguerian M, Chong S, Calvo KR, Deuitch NT, Glushakow-Smith S, Gritsman K, Godley LA, Horwitz MS, Keel S, Castilla LH, Demir E, Mohammed H, Liu P, Agarwal A. Targeting the CD74 signaling axis suppresses inflammation and rescues defective hematopoiesis in RUNX1-familial platelet disorder. Sci Transl Med 2025;17:eadn9832. [PMID: 39772771 PMCID: PMC11912227 DOI: 10.1126/scitranslmed.adn9832] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/11/2024] [Revised: 08/11/2024] [Accepted: 11/03/2024] [Indexed: 01/30/2025]
3
Tanaka Y, Nakanishi Y, Furuhata E, Nakada KI, Maruyama R, Suzuki H, Suzuki T. FLI1 is associated with regulation of DNA methylation and megakaryocytic differentiation in FPDMM caused by a RUNX1 transactivation domain mutation. Sci Rep 2024;14:14080. [PMID: 38890442 PMCID: PMC11189521 DOI: 10.1038/s41598-024-64829-4] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/08/2024] [Accepted: 06/13/2024] [Indexed: 06/20/2024]  Open
4
Jiang YZ, Hu LY, Chen MS, Wang XJ, Tan CN, Xue PP, Yu T, He XY, Xiang LX, Xiao YN, Li XL, Ran Q, Li ZJ, Chen L. GATA binding protein 2 mediated ankyrin repeat domain containing 26 high expression in myeloid-derived cell lines. World J Stem Cells 2024;16:538-550. [PMID: 38817334 PMCID: PMC11135246 DOI: 10.4252/wjsc.v16.i5.538] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 12/26/2023] [Revised: 03/12/2024] [Accepted: 04/12/2024] [Indexed: 05/24/2024]  Open
5
Lee BC, Zhou Y, Bresciani E, Ozkaya N, Dulau-Florea A, Carrington B, Shin TH, Baena V, Syed ZA, Hong SG, Zhen T, Calvo KR, Liu P, Dunbar CE. A RUNX1-FPDMM rhesus macaque model reproduces the human phenotype and predicts challenges to curative gene therapies. Blood 2023;141:231-237. [PMID: 36322931 PMCID: PMC9936307 DOI: 10.1182/blood.2022018193] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/19/2022] [Revised: 10/14/2022] [Accepted: 10/19/2022] [Indexed: 11/06/2022]  Open
6
Lee K, Ahn HS, Estevez B, Poncz M. RUNX1-deficient human megakaryocytes demonstrate thrombopoietic and platelet half-life and functional defects. Blood 2023;141:260-270. [PMID: 36219879 PMCID: PMC9936297 DOI: 10.1182/blood.2022017561] [Citation(s) in RCA: 9] [Impact Index Per Article: 4.5] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/27/2022] [Revised: 09/16/2022] [Accepted: 09/23/2022] [Indexed: 01/24/2023]  Open
7
Zhang S, Qu K, Lyu S, Hoyle DL, Smith C, Cheng L, Cheng T, Shen J, Wang ZZ. PEAR1 is a potential regulator of early hematopoiesis of human pluripotent stem cells. J Cell Physiol 2023;238:179-194. [PMID: 36436185 DOI: 10.1002/jcp.30924] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/27/2022] [Revised: 10/17/2022] [Accepted: 10/28/2022] [Indexed: 11/28/2022]
8
Nations CC, Pavani G, French DL, Gadue P. Modeling genetic platelet disorders with human pluripotent stem cells: mega-progress but wanting more on our plate(let). Curr Opin Hematol 2021;28:308-314. [PMID: 34397590 PMCID: PMC8371829 DOI: 10.1097/moh.0000000000000671] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/25/2022]
9
Li Y, Yang W, Devidas M, Winter SS, Kesserwan C, Yang W, Dunsmore KP, Smith C, Qian M, Zhao X, Zhang R, Gastier-Foster JM, Raetz EA, Carroll WL, Li C, Liu PP, Rabin KR, Sanda T, Mullighan CG, Nichols KE, Evans WE, Pui CH, Hunger SP, Teachey DT, Relling MV, Loh ML, Yang JJ. Germline RUNX1 variation and predisposition to childhood acute lymphoblastic leukemia. J Clin Invest 2021;131:147898. [PMID: 34166225 PMCID: PMC8409579 DOI: 10.1172/jci147898] [Citation(s) in RCA: 19] [Impact Index Per Article: 4.8] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/20/2021] [Accepted: 06/22/2021] [Indexed: 12/31/2022]  Open
10
Borst S, Nations CC, Klein JG, Pavani G, Maguire JA, Camire RM, Drazer MW, Godley LA, French DL, Poncz M, Gadue P. Study of inherited thrombocytopenia resulting from mutations in ETV6 or RUNX1 using a human pluripotent stem cell model. Stem Cell Reports 2021;16:1458-1467. [PMID: 34019812 PMCID: PMC8190596 DOI: 10.1016/j.stemcr.2021.04.013] [Citation(s) in RCA: 14] [Impact Index Per Article: 3.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/25/2020] [Revised: 04/20/2021] [Accepted: 04/21/2021] [Indexed: 12/29/2022]  Open
11
Restoring RUNX1 deficiency in RUNX1 familial platelet disorder by inhibiting its degradation. Blood Adv 2021;5:687-699. [PMID: 33560381 DOI: 10.1182/bloodadvances.2020002709] [Citation(s) in RCA: 7] [Impact Index Per Article: 1.8] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/18/2020] [Accepted: 12/16/2020] [Indexed: 12/15/2022]  Open
12
The Emerging Role of Hematopathologists and Molecular Pathologists in Detection, Monitoring, and Management of Myeloid Neoplasms with Germline Predisposition. Curr Hematol Malig Rep 2021;16:336-344. [PMID: 34028637 DOI: 10.1007/s11899-021-00636-2] [Citation(s) in RCA: 5] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 03/19/2021] [Indexed: 12/12/2022]
13
RUNX-1 haploinsufficiency causes a marked deficiency of megakaryocyte-biased hematopoietic progenitor cells. Blood 2021;137:2662-2675. [PMID: 33569577 DOI: 10.1182/blood.2020006389] [Citation(s) in RCA: 20] [Impact Index Per Article: 5.0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/15/2020] [Accepted: 01/17/2021] [Indexed: 12/18/2022]  Open
14
Bąk A, Skonieczka K, Jaśkowiec A, Junkiert-Czarnecka A, Heise M, Pilarska-Deltow M, Potoczek S, Czyżewska M, Haus O. Searching for germline mutations in the RUNX1 gene among Polish patients with acute myeloid leukemia. Leuk Lymphoma 2021;62:1749-1755. [PMID: 33563056 DOI: 10.1080/10428194.2021.1881503] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 10/22/2022]
15
Reilly A, Doulatov S. Induced pluripotent stem cell models of myeloid malignancies and clonal evolution. Stem Cell Res 2021;52:102195. [PMID: 33592565 PMCID: PMC10115516 DOI: 10.1016/j.scr.2021.102195] [Citation(s) in RCA: 4] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 08/18/2020] [Revised: 01/15/2021] [Accepted: 01/20/2021] [Indexed: 12/13/2022]  Open
16
Yang Y, Li T, Geng Y, Li J. [RUNX1 gene mutations are associated with adverse prognosis of patients with acute myeloidleukemia]. NAN FANG YI KE DA XUE XUE BAO = JOURNAL OF SOUTHERN MEDICAL UNIVERSITY 2020;40:1601-1606. [PMID: 33243739 DOI: 10.12122/j.issn.1673-4254.2020.11.10] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Subscribe] [Scholar Register] [Indexed: 11/24/2022]
17
Donada A, Basso-Valentina F, Arkoun B, Monte-Mor B, Plo I, Raslova H. Induced pluripotent stem cells and hematological malignancies: A powerful tool for disease modeling and drug development. Stem Cell Res 2020;49:102060. [PMID: 33142254 DOI: 10.1016/j.scr.2020.102060] [Citation(s) in RCA: 4] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 03/02/2020] [Revised: 10/09/2020] [Accepted: 10/16/2020] [Indexed: 01/12/2023]  Open
18
Das D, Feuer K, Wahbeh M, Avramopoulos D. Modeling Psychiatric Disorder Biology with Stem Cells. Curr Psychiatry Rep 2020;22:24. [PMID: 32318888 PMCID: PMC8011956 DOI: 10.1007/s11920-020-01148-1] [Citation(s) in RCA: 18] [Impact Index Per Article: 3.6] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 02/06/2023]
19
Olofsen PA, Touw IP. RUNX1 Mutations in the Leukemic Progression of Severe Congenital Neutropenia. Mol Cells 2020;43:139-144. [PMID: 32041395 PMCID: PMC7057833 DOI: 10.14348/molcells.2020.0010] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.6] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/07/2020] [Accepted: 01/08/2020] [Indexed: 12/19/2022]  Open
20
Choi IY, Lim H, Cho HJ, Oh Y, Chou BK, Bai H, Cheng L, Kim YJ, Hyun S, Kim H, Shin JH, Lee G. Transcriptional landscape of myogenesis from human pluripotent stem cells reveals a key role of TWIST1 in maintenance of skeletal muscle progenitors. eLife 2020;9:e46981. [PMID: 32011235 PMCID: PMC6996923 DOI: 10.7554/elife.46981] [Citation(s) in RCA: 33] [Impact Index Per Article: 6.6] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/19/2019] [Accepted: 01/14/2020] [Indexed: 12/15/2022]  Open
21
Thom CS, Chou ST, French DL. Mechanistic and Translational Advances Using iPSC-Derived Blood Cells. JOURNAL OF EXPERIMENTAL PATHOLOGY 2020;1:36-44. [PMID: 33768218 PMCID: PMC7990314 DOI: 10.33696/pathology.1.010] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Download PDF] [Figures] [Subscribe] [Scholar Register] [Indexed: 12/03/2022]
22
Modeling Leukemia with Human Induced Pluripotent Stem Cells. Cold Spring Harb Perspect Med 2019;9:cshperspect.a034868. [PMID: 31451537 DOI: 10.1101/cshperspect.a034868] [Citation(s) in RCA: 5] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/27/2022]
23
Galera P, Dulau-Florea A, Calvo KR. Inherited thrombocytopenia and platelet disorders with germline predisposition to myeloid neoplasia. Int J Lab Hematol 2019;41 Suppl 1:131-141. [PMID: 31069978 DOI: 10.1111/ijlh.12999] [Citation(s) in RCA: 41] [Impact Index Per Article: 6.8] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/02/2019] [Revised: 02/07/2019] [Accepted: 02/10/2019] [Indexed: 12/21/2022]
24
Georgomanoli M, Papapetrou EP. Modeling blood diseases with human induced pluripotent stem cells. Dis Model Mech 2019;12:12/6/dmm039321. [PMID: 31171568 PMCID: PMC6602313 DOI: 10.1242/dmm.039321] [Citation(s) in RCA: 19] [Impact Index Per Article: 3.2] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/14/2022]  Open
25
NOTCHing down a win for megakaryocytes. Blood 2019;131:158-159. [PMID: 29326349 DOI: 10.1182/blood-2017-11-815464] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.2] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 01/09/2023]  Open
26
RUNX1-targeted therapy for AML expressing somatic or germline mutation in RUNX1. Blood 2019;134:59-73. [PMID: 31023702 DOI: 10.1182/blood.2018893982] [Citation(s) in RCA: 71] [Impact Index Per Article: 11.8] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/25/2018] [Accepted: 04/16/2019] [Indexed: 12/22/2022]  Open
27
Saha SK, Saikot FK, Rahman MS, Jamal MAHM, Rahman SMK, Islam SMR, Kim KH. Programmable Molecular Scissors: Applications of a New Tool for Genome Editing in Biotech. MOLECULAR THERAPY. NUCLEIC ACIDS 2019;14:212-238. [PMID: 30641475 PMCID: PMC6330515 DOI: 10.1016/j.omtn.2018.11.016] [Citation(s) in RCA: 27] [Impact Index Per Article: 4.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Subscribe] [Scholar Register] [Received: 05/28/2018] [Revised: 11/23/2018] [Accepted: 11/23/2018] [Indexed: 01/04/2023]
28
Churpek JE, Bresnick EH. Transcription factor mutations as a cause of familial myeloid neoplasms. J Clin Invest 2019;129:476-488. [PMID: 30707109 DOI: 10.1172/jci120854] [Citation(s) in RCA: 44] [Impact Index Per Article: 7.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/13/2022]  Open
29
Elbadry MI, Espinoza JL, Nakao S. Disease modeling of bone marrow failure syndromes using iPSC-derived hematopoietic stem progenitor cells. Exp Hematol 2019;71:32-42. [PMID: 30664904 DOI: 10.1016/j.exphem.2019.01.006] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/24/2018] [Revised: 01/04/2019] [Accepted: 01/15/2019] [Indexed: 01/19/2023]
30
Stem Cell-Derived Platelets. Platelets 2019. [DOI: 10.1016/b978-0-12-813456-6.00066-7] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/26/2022]
31
Kim H, Schaniel C. Modeling Hematological Diseases and Cancer With Patient-Specific Induced Pluripotent Stem Cells. Front Immunol 2018;9:2243. [PMID: 30323816 PMCID: PMC6172418 DOI: 10.3389/fimmu.2018.02243] [Citation(s) in RCA: 5] [Impact Index Per Article: 0.7] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/29/2018] [Accepted: 09/10/2018] [Indexed: 12/13/2022]  Open
32
Li Y, Jin C, Bai H, Gao Y, Sun S, Chen L, Qin L, Liu PP, Cheng L, Wang QF. Human NOTCH4 is a key target of RUNX1 in megakaryocytic differentiation. Blood 2018;131:191-201. [PMID: 29101237 PMCID: PMC5757696 DOI: 10.1182/blood-2017-04-780379] [Citation(s) in RCA: 32] [Impact Index Per Article: 4.6] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/19/2017] [Accepted: 10/13/2017] [Indexed: 12/19/2022]  Open
33
Borst S, Sim X, Poncz M, French DL, Gadue P. Induced Pluripotent Stem Cell-Derived Megakaryocytes and Platelets for Disease Modeling and Future Clinical Applications. Arterioscler Thromb Vasc Biol 2017;37:2007-2013. [PMID: 28982668 PMCID: PMC5675007 DOI: 10.1161/atvbaha.117.309197] [Citation(s) in RCA: 24] [Impact Index Per Article: 3.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/14/2017] [Accepted: 09/21/2017] [Indexed: 12/13/2022]
34
Kanagal-Shamanna R, Loghavi S, DiNardo CD, Medeiros LJ, Garcia-Manero G, Jabbour E, Routbort MJ, Luthra R, Bueso-Ramos CE, Khoury JD. Bone marrow pathologic abnormalities in familial platelet disorder with propensity for myeloid malignancy and germline RUNX1 mutation. Haematologica 2017;102:1661-1670. [PMID: 28659335 PMCID: PMC5622850 DOI: 10.3324/haematol.2017.167726] [Citation(s) in RCA: 60] [Impact Index Per Article: 7.5] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/17/2017] [Accepted: 06/20/2017] [Indexed: 01/20/2023]  Open
35
Hayashi Y, Harada Y, Huang G, Harada H. Myeloid neoplasms with germ line RUNX1 mutation. Int J Hematol 2017;106:183-188. [PMID: 28534116 DOI: 10.1007/s12185-017-2258-5] [Citation(s) in RCA: 23] [Impact Index Per Article: 2.9] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/02/2017] [Accepted: 05/16/2017] [Indexed: 01/23/2023]
36
Ackermann M, Kuhn A, Kunkiel J, Merkert S, Martin U, Moritz T, Lachmann N. Ex vivo Generation of Genetically Modified Macrophages from Human Induced Pluripotent Stem Cells. Transfus Med Hemother 2017. [PMID: 28626364 DOI: 10.1159/000477129] [Citation(s) in RCA: 11] [Impact Index Per Article: 1.4] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 01/02/2023]  Open
37
Hematopoietic transcription factor mutations: important players in inherited platelet defects. Blood 2017;129:2873-2881. [PMID: 28416505 DOI: 10.1182/blood-2016-11-709881] [Citation(s) in RCA: 47] [Impact Index Per Article: 5.9] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/17/2016] [Accepted: 01/26/2017] [Indexed: 01/19/2023]  Open
38
Schlegelberger B, Heller PG. RUNX1 deficiency (familial platelet disorder with predisposition to myeloid leukemia, FPDMM). Semin Hematol 2017. [PMID: 28637620 DOI: 10.1053/j.seminhematol.2017.04.006] [Citation(s) in RCA: 66] [Impact Index Per Article: 8.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/11/2022]
39
Mao GF, Goldfinger LE, Fan DC, Lambert MP, Jalagadugula G, Freishtat R, Rao AK. Dysregulation of PLDN (pallidin) is a mechanism for platelet dense granule deficiency in RUNX1 haplodeficiency. J Thromb Haemost 2017;15:792-801. [PMID: 28075530 PMCID: PMC5378588 DOI: 10.1111/jth.13619] [Citation(s) in RCA: 21] [Impact Index Per Article: 2.6] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/20/2016] [Indexed: 01/01/2023]
40
Role of RUNX1 in hematological malignancies. Blood 2017;129:2070-2082. [PMID: 28179279 DOI: 10.1182/blood-2016-10-687830] [Citation(s) in RCA: 336] [Impact Index Per Article: 42.0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/21/2016] [Accepted: 01/27/2017] [Indexed: 12/23/2022]  Open
41
iPSCs and fibroblast subclones from the same fibroblast population contain comparable levels of sequence variations. Proc Natl Acad Sci U S A 2017;114:1964-1969. [PMID: 28167771 DOI: 10.1073/pnas.1616035114] [Citation(s) in RCA: 48] [Impact Index Per Article: 6.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/22/2022]  Open
42
RUNX1 and CBFβ Mutations and Activities of Their Wild-Type Alleles in AML. ADVANCES IN EXPERIMENTAL MEDICINE AND BIOLOGY 2017;962:265-282. [DOI: 10.1007/978-981-10-3233-2_17] [Citation(s) in RCA: 8] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 02/06/2023]
43
Songdej N, Rao AK. Inherited platelet dysfunction and hematopoietic transcription factor mutations. Platelets 2017;28:20-26. [PMID: 27463948 PMCID: PMC5628047 DOI: 10.1080/09537104.2016.1203400] [Citation(s) in RCA: 10] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/23/2016] [Revised: 05/16/2016] [Accepted: 05/30/2016] [Indexed: 01/19/2023]
44
Paes BCMF, Moço PD, Pereira CG, Porto GS, de Sousa Russo EM, Reis LCJ, Covas DT, Picanço-Castro V. Ten years of iPSC: clinical potential and advances in vitro hematopoietic differentiation. Cell Biol Toxicol 2016;33:233-250. [PMID: 28039590 DOI: 10.1007/s10565-016-9377-2] [Citation(s) in RCA: 9] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/24/2016] [Accepted: 12/18/2016] [Indexed: 01/19/2023]
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Koeffler HP, Leong G. Preleukemia: one name, many meanings. Leukemia 2016;31:534-542. [PMID: 27899806 PMCID: PMC5339433 DOI: 10.1038/leu.2016.364] [Citation(s) in RCA: 41] [Impact Index Per Article: 4.6] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/31/2016] [Revised: 11/11/2016] [Accepted: 11/14/2016] [Indexed: 12/15/2022]
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Ripperger T, Tawana K, Kratz C, Schlegelberger B, Fitzgibbon J, Steinemann D. Clinical utility gene card for: Familial platelet disorder with associated myeloid malignancies. Eur J Hum Genet 2016;24:ejhg2015278. [PMID: 26813945 PMCID: PMC4970691 DOI: 10.1038/ejhg.2015.278] [Citation(s) in RCA: 5] [Impact Index Per Article: 0.6] [Reference Citation Analysis] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/31/2015] [Revised: 11/18/2015] [Accepted: 12/08/2015] [Indexed: 01/16/2023]  Open
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How I diagnose and manage individuals at risk for inherited myeloid malignancies. Blood 2016;128:1800-1813. [PMID: 27471235 DOI: 10.1182/blood-2016-05-670240] [Citation(s) in RCA: 125] [Impact Index Per Article: 13.9] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/18/2016] [Accepted: 07/15/2016] [Indexed: 01/24/2023]  Open
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Daly ME. Transcription factor defects causing platelet disorders. Blood Rev 2016;31:1-10. [PMID: 27450272 DOI: 10.1016/j.blre.2016.07.002] [Citation(s) in RCA: 29] [Impact Index Per Article: 3.2] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/25/2016] [Revised: 06/10/2016] [Accepted: 07/12/2016] [Indexed: 01/19/2023]
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Gene correction in patient-specific iPSCs for therapy development and disease modeling. Hum Genet 2016;135:1041-58. [PMID: 27256364 DOI: 10.1007/s00439-016-1691-5] [Citation(s) in RCA: 28] [Impact Index Per Article: 3.1] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/25/2016] [Accepted: 05/18/2016] [Indexed: 12/20/2022]
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Li W, Morrone K, Kambhampati S, Will B, Steidl U, Verma A. Thrombocytopenia in MDS: epidemiology, mechanisms, clinical consequences and novel therapeutic strategies. Leukemia 2015;30:536-44. [PMID: 26500138 DOI: 10.1038/leu.2015.297] [Citation(s) in RCA: 33] [Impact Index Per Article: 3.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/27/2015] [Accepted: 08/03/2015] [Indexed: 12/14/2022]
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