62nd ASH Annual Meeting and Exposition: 112. Thalassemia and Globin Gene Regulation: Poster III

Oral and Poster Abstracts
Poster

Adult, Anemias, Biological, sickle cell disease, Diseases, Non-Biological, Combinations, thalassemia, Therapies, cellular interactions, Biological Processes, Hemoglobinopathies, gene therapy, Technology and Procedures, erythropoiesis, gene editing, Study Population, Clinically relevant, pharmacology, iron metabolism, pathways

2600

Proof of Concept for the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non–Transfusion-Dependent Thalassemia: Interim Results from an Ongoing, Phase 2, Open-Label, Multicenter Study

Kevin H.M. Kuo, MD, MSc, FRCPC¹, D. Mark Layton, MB BS²^*, Ashutosh Lal, MD³, Hanny Al-Samkari, MD⁴, Feng Tai, PhD⁵^*, Megan Lynch, MSN⁵^*, Katrin Uhlig, MD⁵^* and Elliott P. Vichinsky, MD³

¹University Health Network, Toronto, ON, Canada
²Hammersmith Hospital, Imperial College Healthcare NHS Trust, London, United Kingdom
³UCSF Benioff Children's Hospital Oakland, Oakland, CA
⁴Division of Hematology, Massachusetts General Hospital, Dana-Farber Cancer Institute, Harvard University, Boston, MA
⁵Agios Pharmaceuticals, Inc., Cambridge, MA

2601

Investigating Zeta Globin Gene Expression to Develop a Potential Therapy for Alpha Thalassemia Major

Georgia L. Gregory, BS^1,2,3^*, Beeke Wienert, PhD³^*, Marisa Schwab, MD^1,4^*, Billie Rachael Lianoglou, MS^1,4^*, Roger P. Hollis, PhD⁵^*, Donald B. Kohn, MD⁶, Bruce R. Conklin, MD^3,7,8^* and Tippi C. MacKenzie, MD^1,4,9

¹Department of Surgery, UCSF, San Francisco, CA
²Institute of Human Genetics, UCSF, San Francisco, CA
³Gladstone Institutes, UCSF, San Francisco, CA
⁴Center for Maternal-Fetal Precision Medicine, UCSF, San Francisco, CA
⁵Broad Stem Cell Research Center, UCLA, Los Angeles, CA
⁶Broad Stem Cell Research Center, University of California - Los Angeles, Los Angeles, CA
⁷Innovative Genomics Institute, UCB, Berkeley, CA
⁸Departments of Medicine, Ophthalmology, and Pharmacology, UCSF, San Francisco, CA
⁹Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, UCSF, San Francisco, CA

2602

Mgta-145 / Plerixafor-Mediated HSC Mobilization and Intravenous HDAd5/35++ Vector Injection into Mice Allows for Efficient In Vivo HSC Transduction and Stable Gene Marking in Peripheral Blood Cells of CD46-Transgenic and Thalassemia Mice

Chang Li¹^*, Sucheol Gil²^*, Kevin A. Goncalves, PhD³^*, John C. Davis Jr., MD, MPH, MS³^*, Hans-Peter Kiem, MD PhD⁴ and Andre Lieber, MD, PhD²

¹Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, WA
²University of Washington, Seattle, WA
³Magenta Therapeutics, Cambridge, MA
⁴Fred Hutchinson Cancer Research Center, Seattle, WA

2603

Combinatorial Administration of Pharmacological Inhibitors Targeting DNMT1 and KDM1A Produces Synergistic and Additive Increases in F Retiulocytes and γ-Globin Expression in Normal Baboons

Vinzon Ibanez, B.S.^1,2^*, Kestis Vaitkus²^*, Robert E. Molokie, MD^3,4, Yogenthiran Saunthararajah, MD^5,6 and Donald Lavelle, PhD^2,7

¹Department of Medicine, University of Illinois Chicago, chicago
²Department of Medicine, University of Illinois at Chicago, Jesse Brown VA Medical Center, Chicago, IL
³Jesse Brown VA Medical Center, Chicago, IL
⁴Department of Medicine, Division of Hematology and Oncology, University of Illinois at Chicago, Chicago, IL
⁵Department of Translational Hematology and Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
⁶Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
⁷Department of Medicine, College of Medicine, University of Illinois at Chicago, Chicago, IL

2604

Loss of Alpha Globin Genes in Human Subjects Is Associated with Improved Nitric Oxide-Mediated Vascular Perfusion

Christopher C Denton, MD¹^*, Payal Shah, MS¹^*, Silvie Suriany, MS²^*, Honglei Liu, MS²^*, Wanwara Thuptimdang, MS³^*, John Sunwoo, PhD^3,4^*, Patjanaporn Chalacheva, PhD^3,5^*, Saranya Veluswamy, MD¹^*, Roberta Miyeko Kato, MD⁶^*, John C Wood, MD, PhD²^*, Jon A Detterich, MD²^*, Michael C.K. Khoo, PhD³^* and Thomas D. Coates, MD¹

¹Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, CA
²Cardiology, Children's Hospital Los Angeles, Los Angeles, CA
³Biomedical Engineering, University of Southern California, Los Angeles, CA
⁴Athinoula A. Martinos Center for Biomedical Engineering, Harvard Medical School, Massachusetts General Hospital, Charlestown, MA
⁵Biomedical Engineering, Carnegie Mellon University, Pittsburgh, PA
⁶Pulmonology, Children's Hospital Los Angeles, Los Angeles, CA

2605

Genetic Heterogeneity of KLF1, a Master Regulator of Erythropoiesis, Revealed an Autosomal Recessive Ψβ-Thalassemia and a Very Strong Promoter In Vivo

Laura Grech, PhD, MSc, B.Sc¹^*, Joseph Borg, PhD, MSc, BSc², Ruth Galdies, M.Sc BPharm^1,3^*, Carmen Attard³^*, Christian A. Scerri, MD, PhD^1,3^*, Sjaak Philipsen, PhD⁴^* and Alexander E. Felice^1,3

¹Thalassemia Testing and Hemoglobin Research Laboratory, and The Malta Bio-Bank, Department of Physiology and Biochemistry, University of Malta, Msida, Malta
²Department of Applied Biomedical Science, Faculty of Health Sciences, Mater Dei Hospital, Birkirkara, Malta
³Division of Pathology, Thalassemia and Genetics Clinic, Mater Dei Hospital, Msida, Malta
⁴Department of Cell Biology, Erasmus MC, Rotterdam, Netherlands

2606

Anti-GDF11Treatment of β-Thalassemia Intermedia Mice Does Not Improve Erythropoiesis

Heinrich E Lob, PhD, Leah Kravets, B.S.^*, Lawrence Miloscio, BS^*, Jason Mastaitis, PhD^*, Aris N Economides, PhD^* and Sarah J Hatsell, PhD^*

Regeneron Pharmaceuticals, Inc., Tarrytown, NY

2607

Crispra: A Promising Tool for β-Thalassemia Treatment

Xiaoqing Jia¹^*, Qi Yao²^*, Hui Li³^* and Jieping Chen⁴^*

¹Southwest Hospital，Third Military Medical University(Army Medical University), Chongqing, China
²Southwest Hospital，Third Military Medical University (Army Medical University), Chongqing, China
³Southwest Hospital，Third Military Medical University, Chongqing, China
⁴Southwest Hospital, Third Military Medical University (Army Medical University), Chongqing, China

See more of: Oral and Poster Abstracts

<< Previous Session | Next Session >>

^*signifies non-member of ASH