Targeted gene correction of RUNX1 in induced pluripotent stem cells derived from familial platelet disorder with propensity to myeloid malignancy restores normal megakaryopoiesis

Experimental Hematology

Volumn 43, Issue 10, 2015, Pages 849-857

  Iizuka, Hiromitsu ^a,b   Kagoya, Yuki ^a,b   Kataoka, Keisuke ^a,b   Yoshimi, Akihide ^a,b   Miyauchi, Masashi ^a,b   Taoka, Kazuki ^a,b   Kumano, Keiki ^a,b   Yamamoto, Takashi ^c   Hotta, Akitsu ^b,d   Arai, Shunya ^a,b   Kurokawa, Mineo ^a,b  

^a UNIVERSITY OF TOKYO   (Japan)

^b JAPAN SCIENCE AND TECHNOLOGY AGENCY   (Japan)

^c HIROSHIMA UNIVERSITY   (Japan)

^d KYOTO UNIVERSITY   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; TRANSCRIPTION FACTOR RUNX1; RUNX1 PROTEIN, HUMAN; STOP CODON;

ACUTE MYELOBLASTIC LEUKEMIA; ADULT; ARTICLE; CELL ASSAY; CELL DIFFERENTIATION; CELL MATURATION; CLINICAL FEATURE; COLONY FORMING CELL; CONTROLLED STUDY; DISEASE ASSOCIATION; DNA SEQUENCE; FAMILIAL PLATELET DISORDER; FEMALE; GENE INTERACTION; GENE TARGETING; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; MEGAKARYOPOIESIS; NONSENSE MUTATION; PHENOTYPE; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; RUNX1 GENE; SKIN FIBROBLAST; TALEN GENE; THROMBOCYTE DISORDER; THROMBOCYTOPENIA; BIOSYNTHESIS; BLOOD COAGULATION DISORDERS, INHERITED; BLOOD PLATELET DISORDERS; GENE THERAPY; GENETICS; LEUKEMIA, MYELOID, ACUTE; METABOLISM; PATHOLOGY; PROCEDURES; STOP CODON; THROMBOCYTOPOIESIS;

ADULT; BLOOD COAGULATION DISORDERS, INHERITED; BLOOD PLATELET DISORDERS; CODON, NONSENSE; CORE BINDING FACTOR ALPHA 2 SUBUNIT; DNA, COMPLEMENTARY; FEMALE; GENETIC THERAPY; HUMANS; INDUCED PLURIPOTENT STEM CELLS; LEUKEMIA, MYELOID, ACUTE; THROMBOPOIESIS;

EID: 84942552249     PISSN: 0301472X     EISSN: 18732399     Source Type: Journal    
DOI: 10.1016/j.exphem.2015.05.004     Document Type: Article

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