Abstract:
:In this review, we discuss disease-causing alterations of RUNT-related transcription factor 1 (RUNX1), a master regulator of hematopoietic differentiation. Familial platelet disorder with predisposition to myeloid leukemia (FPDMM) typically presents with (1) mild to moderate thrombocytopenia with normal-sized platelets; (2) functional platelets defects leading to prolonged bleeding; and (3) an increased risk to develop myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), or T-cell acute lymphoblastic leukemia (T-ALL). Hematological neoplasms in carriers of a germline RUNX1 mutation need additional secondary mutations or chromosome aberrations to develop. If a disease-causing mutation is known in the family, it is important to prevent hematopoietic stem cell transplantation from a sibling or other relative carrying the familial mutation. First experiments introducing a wild-type copy of RUNX1 into induce pluripotent stem cells (iPSC) lines from patients with FPDMM appear to demonstrate that by gene correction reversal of the phenotype may be possible.
journal_name
Semin Hematoljournal_title
Seminars in hematologyauthors
Schlegelberger B,Heller PGdoi
10.1053/j.seminhematol.2017.04.006subject
Has Abstractpub_date
2017-04-01 00:00:00pages
75-80issue
2eissn
0037-1963issn
1532-8686pii
S0037-1963(17)30047-1journal_volume
54pub_type
杂志文章,评审abstract::Since warfarin remains the predominate drug administered for long-term anticoagulation, optimizing therapy for maximum antithrombotic effect with minimal bleeding risk continues to challenge clinicians. Genetic differences exist that affect an individual's response to warfarin. The clinician can use genetic informatio...
journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/shem.2002.34089
更新日期:2002-07-01 00:00:00
abstract::High-dose chemoradiotherapy with allogeneic bone marrow or peripheral blood stem cell transplantation (SCT) is a potentially curative treatment for advanced or poor-prognosis hematological malignancies. This procedure was initially considered as a means to deliver supralethal doses of chemotherapy and radiation for th...
journal_title:Seminars in hematology
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
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更新日期:2003-04-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
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更新日期:2010-01-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2008.03.002
更新日期:2008-04-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章
doi:10.1053/j.seminhematol.2020.12.002
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1993-10-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2009.02.003
更新日期:2009-04-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1999-10-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章
doi:10.1053/j.seminhematol.2003.10.001
更新日期:2004-01-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1998-07-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/shem.2003.50046
更新日期:2003-04-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
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更新日期:2020-01-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
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更新日期:2018-07-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1983-10-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/shem.2003.50002
更新日期:2003-01-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1053/j.seminhematol.2017.06.003
更新日期:2017-07-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
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更新日期:2007-04-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1016/s0037-1963(01)90131-3
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1997-10-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1997-01-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:10.1016/s0037-1963(01)90052-6
更新日期:2001-04-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 临床试验,杂志文章,评审
doi:
更新日期:1996-10-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 临床试验,杂志文章
doi:
更新日期:1997-07-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
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更新日期:2005-07-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
更新日期:1999-01-01 00:00:00
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journal_title:Seminars in hematology
pub_type: 杂志文章,评审
doi:
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