Physiologic Expression of Sf3b1(K700E) Causes Impaired Erythropoiesis, Aberrant Splicing, and Sensitivity to Therapeutic Spliceosome Modulation.

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第一作者： Esther A,Obeng

第一单位： Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA 02115, USA; Division of Hematology/Oncology, Department of Medicine, Boston Children's Hospital, Boston, MA 02115, USA; Division of Hematology, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA.

作者： Esther A,Obeng ^[1] ; Ryan J,Chappell ^[2] ; Michael,Seiler ^[3] ; Michelle C,Chen ^[2] ; Dean R,Campagna ^[4] ; Paul J,Schmidt ^[4] ; Rebekka K,Schneider ^[2] ; Allegra M,Lord ^[2] ; Lili,Wang ^[5] ; Rutendo G,Gambe ^[5] ; Marie E,McConkey ^[2] ; Abdullah M,Ali ^[6] ; Azra,Raza ^[6] ; Lihua,Yu ^[3] ; Silvia,Buonamici ^[3] ; Peter G,Smith ^[3] ; Ann,Mullally ^[7] ; Catherine J,Wu ^[8] ; Mark D,Fleming ^[4] ; Benjamin L,Ebert ^[9]

作者单位： Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA 02115, USA; Division of Hematology/Oncology, Department of Medicine, Boston Children's Hospital, Boston, MA 02115, USA; Division of Hematology, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA. ^[1] Division of Hematology, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA. ^[2] H3 Biomedicine, Inc., Cambridge, MA 03129, USA. ^[3] Department of Pathology, Boston Children's Hospital, Boston, MA 02115, USA. ^[4] Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02115, USA. ^[5] Division of Hematology/Oncology, Columbia University Medical Center, New York, NY 10027, USA. ^[6] Division of Hematology, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA. ^[7] Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02115, USA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA. ^[8] Division of Hematology, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA. Electronic address: bebert@partners.org. ^[9]