62nd ASH Annual Meeting and Exposition: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Mechanisms, Diagnosis and Treatment of Inherited

Oral and Poster Abstracts
Oral

Adult, Anemias, HSCs, Biological, Diseases, Animal models, aplastic anemia, cell division, iPSCs, cell regulation, Therapies, Genetic Disorders, red blood cells, blood banking, Biological Processes, Technology and Procedures, cell expansion, gene therapy, Cell Lineage, gene editing, Study Population, erythropoiesis, genomics, Clinically relevant, hematopoiesis, imaging, flow cytometry, stem cells, NGS, molecular interactions, pathogenesis, pathways, proteomics

Moderators:

Roberta Russo, CEINGE Biotecnologie Avanzate s.c.a.r.l. University Federico II of Naples and John G. Quigley,

Disclosures:

No relevant conflicts of interest to declare.

9:30 AM

Modelling Congenital Dyserythropoietic Anemia Type II through Gene Editing in Hematopoietic Stem and Progenitor Cells

Mercedes Dessy-Rodriguez^1,2^*, Sara Fañanas-Baquero^1,2^*, Veronica Venturi³^*, Salvador Payán-Pernía⁴^*, Cristian Tornador⁵^*, Gonzalo Hernandez³^*, Mayka Sanchez^5,6^*, José C Segovia, PhD^1,7^* and Oscar Quintana Bustamante, PhD^1,7^*

¹Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain
²Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez. (IIS-FJD, UAM), Madrid, Spain
³Universitat Internacional de Catalunya, Barcelona, Spain
⁴National Reference Centre (CSUR) for Hereditary Red Blood Cell Disorders, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain
⁵Bloodgenetics, Barcelona, Spain
⁶Universitat Internacional De Catalunya, Sant Cugat Del Vallès-Barcelona, Spain
⁷Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD, UAM), Madrid, Spain

9:45 AM

Novel Fluorescent Timer Tool Enables Characterization of Erythropoietic Differentiation Based on Differential Cell Cycling Speeds

Susree Modepalli, PhD¹^*, Anna Eastman, PhD^2,3^*, Chloe Shaw⁴^*, Shangqin Guo, PhD^2,3^*, Shilpa M. Hattangadi, MD⁵ and Gary M. Kupfer, MD⁶

¹Department of Oncology, Georgetown University, Washington, DC
²Department of Cell Biology, Yale University, New Haven, CT
³Yale Stem Cell Center, Yale University, New Haven, CT
⁴George Washington University, Washington, DC
⁵NIH, Washington, DC
⁶Department of Oncology, Georgetown University, washington, DC

10:00 AM

Mutation of EPO 5`UTR Facilitates Interaction with HIF2 and Causes Autosomal Dominant Erythrocytosis

Lucie Lanikova, PhD¹, Jihyun Song, PhD², Olga Babosova, PhD¹^*, Linda Berkova, MSc.¹^*, Vladimir Korinek, PhD¹^* and Josef T. Prchal, MD³

¹Department of Cell and Developmental Biology, Institute of Molecular Genetics of the Czech Academy of Sciences, Prague, Czech Republic
²Division of Hematology, University of Utah, Salt Lake City, UT
³Division of Hematology and Hematologic Malignancies, Huntsman Cancer Institute, University of Utah, Salt Lake City, UT

10:15 AM

The Pyruvate Kinase Activator Mitapivat Ameliorates Anemia and Prevents Iron Overload in a Mouse Model of Hereditary Spherocytosis

Alessandro Mattè, BSc, PhD¹^*, Wilson Anand¹^*, Enrica Federti, PhD¹^*, Charles Kung, PhD²^*, Penelope A. Kosinski, MS²^*, Veronica Riccardi¹^*, Iana Iatcenko¹^*, Lenny Dang, PhD²^*, Christophe Leboeuf³^*, Anne Janin, PhD³^*, Carlo Brugnara, MD⁴, Mohandas Narla, DSc⁵ and Lucia De Franceschi, MD¹

¹University of Verona and AOUI Verona, Verona, Italy
²Agios Pharmaceuticals, Inc., Cambridge, MA
³University Diderot of Paris, Paris, France
⁴Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School, Boston, MA
⁵Laboratory of Red Cell Physiology, New York Blood Center, New York, NY

10:30 AM

Correction of the Energetic Defects in Pyruvate Kinase Deficiency through Genome Editing in Hematopoietic Stem and Progenitor Cells

Sara Fañanas-Baquero^1,2^*, Oscar Quintana Bustamante, PhD^1,3^*, Daniel P. Dever, PhD⁴^*, Omaira Alberquilla²^*, Rebeca Sanchez-Dominguez, PhD^1,2^*, Joab Camarena, BS⁴^*, Rolf Turk⁵^*, José Luis López Lorenzo, MD⁶^*, Paola Bianchi, BSc, PhD⁷, Mark Behlke, MD, PhD⁵^*, Juan A Bueren, PhD^3,8^*, Matt Porteus, MD, PhD⁴ and José C Segovia, PhD^1,3^*

¹Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain
²Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez. (IIS-FJD, UAM), Madrid, Spain
³Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD, UAM), Madrid, Spain
⁴Department of Pediatrics, Stanford University School of Medicine, Stanford, CA
⁵Integrated DNA Technologies, Coralville, IA
⁶Hospital Universitario Fundación Jiménez Díaz- IISFJD-UAM, Madrid, Spain
⁷Ospedale Maggiore Policlinico di Milano, Foundation IRCCS Ca' Granda,, Milano, Italy
⁸Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Valencia, Spain

10:45 AM

Characteristics of in Vitro Differentiated Erythrocytes Derived from Human Bmi-1 Extensively Expanded Erythroblasts (E3)

Moira M. Lancelot, MD^1,2, Natia Saakadze, MSc³^*, Jiusheng Deng, PhD¹^*, Sean R. Stowell, MD, PhD⁴, Ryan Jajosky, MD⁵^*, Linzhao Cheng, PhD⁶, Senquan Liu⁷^* and John D Roback, MD, PhD¹^*

¹Center for Transfusion and Cellular Therapies, Department of Pathology and Laboratory Medicine, Emory University School of Medicine, Atlanta, GA
²Emory University Hospital Blood Bank, Atlanta, GA
³Center for Transfusion and Cellular Therapies, Department of Pathology and Laboratory Medicine, Atlanta, GA
⁴Center for Transfusion Medicine and Cellular Therapies, Department of Laboratory Medicine and Pathology, Emory University School of Medicine, Atlanta, GA
⁵Department of Pathology and Laboratory Medicine, Emory University, Atlanta, GA
⁶Division of Hematology, Johns Hopkins University School of Medicine, Baltimore, MD
⁷Division of Hematology, Johns Hopkins University School Of Medicine, Batimore, MD

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^*signifies non-member of ASH

101.O2.6 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Mechanisms, Diagnosis and Treatment of Inherited