ASH News Daily 2015 - Day 3 - (Page A-1)

ASH NEWS DAILY ® 57th Annual Meeting of the American Society of Hematology Issue 3, Section A Monday, December 7, 2015 Orlando, FL Read this issue online at www.hematology.org/ashnewsdaily2015_monday Follow us on Twitter using #ASH15 SCHEDULE 9:00 - 10:00 a.m. E. Donnall Thomas Lecture Orange County Convention Center (Hall D) 10:30 a.m. - 12:00 noon Spotlight Sessions: Beyond Transcription: Translational Control of Gene Expression in Development and Disease Orange County Convention Center (W314) Optimal Delivery of the Neonate with a Bleeding Disorder Orange County Convention Center (W315) Infections and the Blood Supply Orange County Convention Center (W312) 12:15 - 1:15 p.m. Featured Topic Discussion: Precision Therapy Orange County Convention Center (Tangerine 1 - WF1) 12:15 - 1:15 p.m. Meet the Blood Editors Orange County Convention Center (W202A, W202C) 1:30 - 2:30 p.m. Ernest Beutler Lecture Orange County Convention Center (Hall D) 2:45 - 4:15 p.m. Spotlight Sessions: Challenging Anticoagulation Scenarios Orange County Convention Center (W315) Addressing Severe Thrombocytopenia in the Care of Patients with Myelodysplastic Syndromes and Other Hematological Malignancies Orange County Convention Center (W312) Plasma Cell Disorders Other Than Myeloma Orange County Convention Center (W314) 4:30 - 6:30 p.m. Special Symposium on the Basic Science of Hemostasis and Thrombosis Orange County Convention Center (Tangerine 1 - WF1) IN THIS SECTION Hemophilia A-2 Sickle Cell A-4 MPNs A-10 Hemostasis and Thrombosis A-16 Splice Splice Baby BY JEANNE PALMER, MD C hances are, if you have attended any sessions on myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML), you have heard about somatic mutations of spliceosome machinery. The discovery of somatic mutations has rocked the world of hematology, providing novel prognostic information and insight to the pathophysiology of these devastating diseases. This year, one of those mutations has been shown to be a therapeutic target, which earned Dr. Stanley ChunWei Lee and his colleagues a spot in Sunday's plenary session. In their presentation, Dr. Lee and colleagues from several institutions discussed their data using E7107, a novel agent that modulates spliceosome function in AML with spliceosome mutations. Spliceosomes play an important role in cell biology. They help splice together the correct sequence of mRNA to generate proteins vital Dr. Stanley Chun-Wei Lee for cellular processes. Recent studies have shown that there is a high incidence of mutations in the genes for spliceosomes, occurring in 60 to 65 percent of cases of MDS, and in 20 percent of cases of AML. These mutations preferentially affect 3′ premRNA splicing machinery, including SF3B1 (splicing factor 3b subunit 1), SRSF2 (serine/arginine-rich splicing factor 2), and U2AF1 (U2 small nuclear RNA auxiliary factor 1). Research has associated mutations with a variety of outcomes. For example, SF3B1 has been associated with a favorable prognosis and is frequently »» SPLICE Page A-6 Bullseye! Targeted Genetic and Epigenetic Therapies Hit the Mark BY MARK CHAO, MD, PHD, AND MICHAELA LIEDTKE, MD T argeted therapies have never been closer to hitting their mark in hematologic diseases than they are today. The last decade has witnessed unprecedented discovery of disease-related germline and somatic mutations that have provided novel insights into the genetic basis of hematologic disorders. Furthermore, it has become clear that many of these mutations target genes that influence DNA methylation and chromatin modifications and result in unique epigenetic alterations. However, until recently, therapies that target these genetic and epigenetic abnormalities have been scarce. This year's Presidential Symposium will showcase three experts highlighting recent insights in the genome and epigenome of hematologic disorders and show us how therapeutic targeting of associated abnormalities have achieved success in both malignant and nonmalignant hematologic diseases. Dr. Mignon Loh, chief of the Division of Pediatric Hematology/ Oncology at the University of California San Francisco, will begin by discussing the genetic disposition of juvenile myelomonocytic leukemia, including how the discovery of both germline and acquired mutations has elucidated an in-depth understanding of disease initiation and evolution. She will also discuss how these understandings extend beyond this leukemic subtype and »» SYMPOSIUM Page A-6 http://www.hematology.org/ashnewsdaily2015_monday

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