-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

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

Symposia: Thalassemia and Globin Gene Regulation Program: Oral and Poster Abstracts
Type: Oral
Hematology Disease Topics & Pathways:
Biological therapies, adult, Non-Biological therapies, Thalassemia, Hemoglobinopathies, Diseases, Gene Therapy, Therapies, Pharmacology, Technology and Procedures, gene editing, Study Population, Human, Animal model
Sunday, December 10, 2023: 4:30 PM-6:00 PM
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

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

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, MD2,3,4,5

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

Yu Wang1*, Lei Yu1*, Greggory Myers, B.S.1, Sharon A Singh, MD1,2 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

Ginette Balbin-Cuesta, MD, PhD1,2, Greggory Myers, B.S.3, Claire Kerpet, B.S.4*, Beth McGee5*, Katherine Rybkin6*, Zesen Lin, BS7, Claire Drysdale, BSc1,3, 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

Pinyaphat Khamphikham, PhD1,2*, 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

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

*signifies non-member of ASH