65th ASH Annual Meeting & Exposition: 112. Thalassemia and Globin Gene Regulation: Finding New Pathways to Treat Thalassemia

Symposia: Thalassemia and Globin Gene Regulation Oral and Poster Abstracts
Oral

Biological therapies, adult, Non-Biological therapies, Thalassemia, Hemoglobinopathies, Diseases, Gene Therapy, Therapies, Pharmacology, Technology and Procedures, gene editing, Study Population, Human, Animal model

Room 31 (San Diego Convention Center)

Moderators:

James J Bieker, PhD, Mount Sinai School of Medicine and Kevin H.M. Kuo, MD, FRCPC, MSc, University of Toronto

Disclosures:

No relevant conflicts of interest to declare.

4:30 PM

553

Selective Globin Gene Regulation By the Non-Canonical Baf Chromatin Remodeling Complex

Sindhu Billakanti¹^*, Vanessa Shehu¹^*, Kathryn M. Farrell¹^*, Osheiza Abdulmalik, DVM¹^*, Belinda M. Giardine²^*, Cheryl A. Keller, PhD²^*, Ross C. Hardison, PhD²^*, Junwei Shi, PhD³^*, Gerd A. Blobel, MD, PhD¹ and Eugene Khandros, MD, PhD¹

¹Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
²Department of Biochemistry and Molecular Biology, The Pennsylvania State University, University Park, PA
³Department of Cancer Biology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA

4:45 PM

554

Tetramer Formation of BCL11A Is Required for Both Protein Stability and Fetal Hemoglobin Silencing

Ge Zheng¹^*, Stuti Mehta, PhD¹^*, Maolu Yin, PhD¹^*, Linus Marx, M.Sc.¹^*, Altantsetseq Buyanbat¹^*, Karin Tenglin¹^*, Benjamin Sewell-Grossman¹^*, Jiyoung Hong, PhD¹^* and Stuart H Orkin, MD^2,3,4,5

¹Dana Farber/Boston Children's Hospital Cancer and Blood Disorders Center, Boston, MA
²Dana-Farber Boston Children’s Cancer and Blood Disorders Center, Boston, MA
³Dana-Farber Cancer Institute, Inc., Boston, MA
⁴Howard Hughes Medical Institute, Boston
⁵Harvard Medical School, Boston, MA

5:00 PM

555

Direct and Indirect Functions of TR4 in the Control of Fetal Hemoglobin Silencing

Yu Wang¹^*, Lei Yu¹^*, Greggory Myers, B.S.¹, Sharon A Singh, MD^1,2 and James Engel, Ph.D.¹

¹Department of Cell and Developmental Biology, University of Michigan Medical School, Ann Arbor, MI
²Department of Pediatrics, University of Michigan, Ann Arbor, MI

5:15 PM

556

The CDK-Inhibitor p27^Kip1 Regulates Fetal Hemoglobin Expression

Ginette Balbin-Cuesta, MD, PhD^1,2, Greggory Myers, B.S.³, Claire Kerpet, B.S.⁴^*, Beth McGee⁵^*, Katherine Rybkin⁶^*, Zesen Lin, BS⁷, Claire Drysdale, BSc^1,3, Ann Friedman, B.S.⁴^*, Sharon A Singh, MD⁸, Lei Yu⁴^*, Ayse Bilge Ozel, M.S., Ph.D.⁹^* and Rami Khoriaty, M.D.^3,4,10,11

¹Medical Scientist Training Program, University of Michigan Medical School, Ann Arbor, MI
²Cellular and Molecular Biology Program, University of Michigan Medical School, Northville, MI
³Department of Cell and Developmental Biology, University of Michigan Medical School, Ann Arbor, MI
⁴Department of Internal Medicine, University of Michigan Medical School, Ann Arbor, MI
⁵Howard Hughes Medical Institute, Ann Arbor, MI
⁶College of Literature, Science, and the Arts, University of Michigan, Ann Arbor, MI
⁷Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI
⁸Department of Pediatrics, University of Michigan, Ann Arbor, MI
⁹Department of Human Genetics, University of Michigan, Ann Arbor, MI
¹⁰Cellular and Molecular Biology Program, University of Michigan Medical School, Ann Arbor, MI
¹¹University of Michigan Rogel Cancer Center, Ann Arbor, MI

5:30 PM

557

Pharmacologic Inhibition of XPO1 By Selinexor Improves Late-Stage Erythropoiesis in Severely Affected β⁰-Thalassemia/Hemoglobin E

Pinyaphat Khamphikham, PhD^1,2^*, Adisak Tantiworawit, MD³ and Songyot Anuchapreeda, PhD¹^*

¹Division of Clinical Microscopy, Department of Medical Technology, Faculty of Associated Medical Sciences, Chiang Mai University, Chiang Mai, Thailand
²Hematology and Health Technology Research Center, Faculty of Associated Medical Sciences, Chiang Mai University, Chiang Mai, Thailand
³Division of Hematology, Department of Internal Medicine, Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand

5:45 PM

558

Iron Restriction Alleviates β-Thalassemia By Stimulating ULK1-Mediated Autophagy of Free α-Globin

Alfonso G. Fernandez, PhD¹^*, Georgios E. Christakopoulos, MD², Dudley E. Michael³^*, Razan B. A. Sweileh³^*, Rahul Telange, MSc, BS³^*, Heather Sheppard, DVM, PhD⁴^*, Yu Yao, MD³^*, Jingjing Zhang³^*, Vania Manolova, PhD⁵^*, Elizabeta Nemeth, PhD⁶, Tomas Ganz, MD, PhD⁷ and Mitchell Weiss, MD, PhD³

¹Dept. of Hematology, St Jude Children's Research Hospital, Memphis, TN
²Hematology, St Jude Children's Research Hospital, Memphis, TN
³Dept. of Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁴Comparative Pathology Core, St. Jude Children's Research Hospital, Memphis, TN
⁵CSL Vifor, St. Gallen, Switzerland
⁶Center for Iron Disorders, University of California, Los Angeles, Los Angeles, CA
⁷Center for Iron Disorders, David Geffen School of Medicine at UCLA, Los Angeles, CA

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^*signifies non-member of ASH

112.O1.6 112. Thalassemia and Globin Gene Regulation: Finding New Pathways to Treat Thalassemia