Phase I First-in-Human Dose Escalation Study of the oral SF3B1 modulator H3B-8800 in myeloid neoplasms

Dimeric ferrochelatase bridges ABCB7 and ABCB10 homodimers in an architecturally defined molecular complex required for heme biosynthesis

(2019) Nunziata Maio et al.   HAEMATOLOGICA

Structural complementarity facilitates E7820-mediated degradation of RBM39 by DCAF15

(2019) Tyler B. Faust et al.   Nature Chemical Biology

Chronic Myelomonocytic leukemia: 2020 update on diagnosis, risk stratification and management

(2019) Mrinal M. Patnaik et al.   AMERICAN JOURNAL OF HEMATOLOGY

Impact of spliceosome mutations on RNA splicing in myelodysplasia: dysregulated genes/pathways and clinical associations

(2018) Andrea Pellagatti et al.   BLOOD

The cryo-EM structure of the SF3b spliceosome complex bound to a splicing modulator reveals a pre-mRNA substrate competitive mechanism of action

(2018) Lorenzo I. Finci et al.   GENES & DEVELOPMENT

H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers

(2018) Michael Seiler et al.   NATURE MEDICINE

Aberrant splicing and defective mRNA production induced by somatic spliceosome mutations in myelodysplasia

(2018) Yusuke Shiozawa et al.   Nature Communications

Modeling SF3B1 Mutations in Cancer: Advances, Challenges, and Opportunities

(2016) Daichi Inoue et al.   CANCER CELL

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

(2016) Esther A. Obeng et al.   CANCER CELL

Modulation of splicing catalysis for therapeutic targeting of leukemia with mutations in genes encoding spliceosomal proteins

(2016) Stanley Chun-Wei Lee et al.   NATURE MEDICINE

An international consortium proposal of uniform response criteria for myelodysplastic/myeloproliferative neoplasms (MDS/MPN) in adults

(2015) M. R. Savona et al.   BLOOD

Epidemiology and Clinical Significance of Secondary and Therapy-Related Acute Myeloid Leukemia: A National Population-Based Cohort Study

(2015) Lene Sofie Granfeldt Østgård et al.   JOURNAL OF CLINICAL ONCOLOGY

Graphical representation of clinical outcomes for patients with myelodysplastic syndromes

(2015) David P. Steensma   LEUKEMIA & LYMPHOMA

Molecular pathogenesis of atypical CML, CMML and MDS/MPN-unclassifiable

(2014) Katerina Zoi et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

TMEM14C is required for erythroid mitochondrial heme metabolism

(2014) Yvette Y. Yien et al.   JOURNAL OF CLINICAL INVESTIGATION

Disruption of SF3B1 results in deregulated expression and splicing of key genes and pathways in myelodysplastic syndrome hematopoietic stem and progenitor cells

(2014) H Dolatshad et al.   LEUKEMIA

A phase I, open-label, single-arm, dose-escalation study of E7107, a precursor messenger ribonucleic acid (pre-mRNA) splicesome inhibitor administered intravenously on days 1 and 8 every 21 days to patients with solid tumors

(2013) D. S. Hong et al.   INVESTIGATIONAL NEW DRUGS

Mutations affecting mRNA splicing define distinct clinical phenotypes and correlate with patient outcome in myelodysplastic syndromes

(2012) F. Damm et al.   BLOOD

Mutations in the spliceosome machinery, a novel and ubiquitous pathway in leukemogenesis

(2012) H. Makishima et al.   BLOOD

Revised International Prognostic Scoring System for Myelodysplastic Syndromes

(2012) P. L. Greenberg et al.   BLOOD

Splicing factor mutations in myelodysplasia

(2012) Seishi Ogawa   INTERNATIONAL JOURNAL OF HEMATOLOGY

Clinical significance of SF3B1 mutations in myelodysplastic syndromes and myelodysplastic/myeloproliferative neoplasms

(2011) L. Malcovati et al.   BLOOD

The anti-tumor drug E7107 reveals an essential role for SF3b in remodeling U2 snRNP to expose the branch point-binding region

(2011) E. G. Folco et al.   GENES & DEVELOPMENT

Frequent pathway mutations of splicing machinery in myelodysplasia

(2011) Kenichi Yoshida et al.   NATURE

Recurrent mutations in the U2AF1 splicing factor in myelodysplastic syndromes

(2011) Timothy A Graubert et al.   NATURE GENETICS

SomaticSF3B1Mutation in Myelodysplasia with Ring Sideroblasts

(2011) E. Papaemmanuil et al.   NEW ENGLAND JOURNAL OF MEDICINE