57th ASH Annual Meeting and Exposition (December 5-8, 2015): 112. Thalassemia and Globin Gene Regulation: Understanding and Manipulating Globin Gene Regulation

Thalassemia and Globin Gene Regulation Oral and Poster Abstracts
Oral

W414AB, Level 4 (Orange County Convention Center)

Moderators:

Swee Lay Thein, MD, DSc, National Institutes of Health and Jian Xu, Ph.D., University of Texas Southwestern Medical Center

Disclosures:

No relevant conflicts of interest to declare.

2:45 PM

3372

Deletional HPFH Vs. Delta Beta Thalassemia: Closing in on a Possible Hb F Silencer Location

Molly Susan Hein, BS¹^*, Kenneth C Swanson, MT(ASCP)¹^*, Patrick A Lundquist, MB(ASCP), CG(ASCP)²^*, Joella A Yungerberg, MLS(ASCP)¹^*, Lea M Coon, MS¹^*, Brian D Dawson, PhD²^*, Dragan Jevremovic, MD PhD¹^*, Andre M Oliveira, MD, PhD²^*, James D Hoyer, MD¹ and Jennifer L Oliveira, MD¹

¹Hematopathology and Metabolic Hematology Laboratory, Mayo Clinic, Rochester, MN
²Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN

3:00 PM

638

Crispr-Cas9 Saturating Mutagenesis Reveals an Achilles Heel in the BCL11A Erythroid Enhancer for Fetal Hemoglobin Induction (by Genome Editing)

Daniel E. Bauer, M.D., Ph.D.¹, Matthew C. Canver, B.S.¹^*, Elenoe C. Smith, Ph.D.²^*, Falak Sher, Ph.D.¹^*, Luca Pinello, Ph.D.³^*, Neville E. Sanjana, Ph.D.⁴^*, Ophir Shalem, Ph.D.⁴^*, Diane D. Chen, B.S.⁵^*, Patrick G. Schupp, B.S.⁵^*, Divya S. Vinjamur, Ph.D.⁶^*, Sara P. Garcia, Ph.D.³^*, Sidinh Luc, Ph.D.², Ryo Kurita, Ph.D.⁷^*, Yukio Nakamura, M.D., Ph.D.⁷^*, Yuko Fujiwara, Ph.D.⁸^*, Takahiro Maeda, M.D., Ph.D.⁹, Guo-Cheng Yuan, Ph.D.³^*, Guillaume Lettre, Ph.D.¹⁰^*, Feng Zhang, Ph.D.¹¹^* and Stuart H. Orkin, M.D.¹²

¹Pediatric Hematology-Oncology, Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA
²Pediatric Hematology/Oncology, Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA
³Biostatistics and Computational Biology, Dana-Farber Cancer Institute, Harvard School of Public Health, Boston, MA
⁴Broad Institute of MIT and Harvard, Cambridge, MA
⁵Boston Children's Hospital, Boston, MA
⁶Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA
⁷Cell Engineering Division, RIKEN BioResource Center, Tsukuba, Japan
⁸Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Medical School, HHMI, Boston, MA
⁹Division of Hematology, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA
¹⁰Montreal Heart Institute, Universite de Montreal, Montreal, QC, Canada
¹¹Broad Institute of MIT and Harvard, Massachusetts Institute of Technology, Cambridge, MA
¹²Department of Pediatric Oncology, Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Medical School, HHMI, Boston, MA

3:15 PM

639

IGF2BP1 Reverses Hemoglobin Switching in Adult Erythroblasts

Laxminath Tumburu^*, Colleen Byrnes, MS^*, Y. Terry Lee^*, Jaira F. de Vasconcellos^*, Antoinette Rabel^* and Jeffery L. Miller, MD

Molecular Genomics and Therapeutics Section, Molecular Medicine Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD

3:30 PM

640

Genome Editing Recreates Hereditary Persistence of Fetal Hemoglobin in Primary Human Erythroblasts

Elizabeth Traxler^1,2^*, Yu Yao, MD²^*, Chunliang Li³^*, Jeremy Grevet¹^*, Peng Huang, Phd⁴^*, Shaela Wright³^*, Gerd A. Blobel, MD, PhD⁵ and Mitchell J. Weiss, MD, PhD²

¹Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA
²Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
³St. Jude Children's Research Hospital, Memphis, TN
⁴Children's Hospital of Philadelphia, Philadelphia, PA
⁵Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA

3:45 PM

641

ASH1L: A Novel Beta-Globin Gene Regulator in Humans?

Amandine Breton, PhD¹^*, Laura Sonzogni, PhD¹^*, Andria Theodorou, PhD¹^*, Suleyman Aktuna, PhD¹^*, Stephan Menzel, PhD¹^*, Frank Grosveld, PhD²^*, Sjaak Philipsen, PhD²^* and Swee Lay Thein, MD, DSc^3,4

¹Molecular Haematology, Kings College London, London, United Kingdom
²Cell Biology, Erasmus MC, Rotterdam, Netherlands
³Molecular Haematology, Division of Cancer Studies, King's College London, London, United Kingdom
⁴National Heart, Lung and Blood Institute, Sickle Cell Branch, National Institutes of Health, Bethesda, MD

4:00 PM

642

The LSD1 Inhibitor RN-1 Recapitulates the Fetal Pattern of Hemoglobin Synthesis in Anemic and Normal, Non-Anemic Baboons

Angela Rivers, MD, PhD¹, Kestis Vaitkus²^*, Vinzon Ibanez²^*, Maria Armila Ruiz²^*, Ramaswamy Jagadeeswaran, PhD¹^*, Joseph DeSimone, PhD² and Donald Lavelle, PhD²

¹Department of Pediatrics, University of Illinois at Chicago, Jesse Brown VA Medical Center, Chicago, IL
²Department of Medicine, University of Illinois at Chicago, Jesse Brown VA Medical Center, Chicago, IL

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^*signifies non-member of ASH

112.O1.6 112. Thalassemia and Globin Gene Regulation: Understanding and Manipulating Globin Gene Regulation