66th ASH Annual Meeting: 321. Coagulation and Fibrinolysis: Basic and Translational. A Coagulation Grab Bag

Symposia: Coagulation and Fibrinolysis: Basic and Translational Oral and Poster Abstracts
Oral

Research, Fundamental Science, Bleeding and Clotting, Bleeding disorders, Translational Research, Hemophilia, Genetic Disorders, Diseases, Thrombotic disorders, VWD, Biological Processes, Molecular biology

Room 25 (San Diego Convention Center)

Moderators:

Radoslaw Kaczmarek, Indiana University School of Medicine and Carlos Bravo-Perez, MD, ARRAY(0xe7858fc)

Disclosures:

Kaczmarek: Bayer: Consultancy, Honoraria, Research Funding; Pfizer: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; BioMarin: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Spark: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Novo Nordisk: Consultancy, Honoraria.

In this session, authors present novel therapuetic approaches, new disease models for researchers, fresh insights into pathophysiology, and new mechanisms of disease.

9:30 AM

427

Enhanced Hemostatic Efficacy of a Membrane and Factor IXa Binding Antibody Fragment with Cellular Recyclability in Hemophilia A

Shekhar Kumar, PhD^1,2 and Sriram Krishnaswamy, PhD^1,2

¹Research Institute, Children's Hospital of Philadelphia, Philadelphia, PA
²Pediatrics, University of Pennsylvania, Philadelphia, PA

9:45 AM

428

Cryoem Structures and Conformational Landscapes of Activated Blood Coagulation Factor VIII and the Intrinsic Tenase Complex

Kenneth C Childers¹^*, Omar Davulcu²^*, Carmen Helena Coxon, PhD³^*, Pete Lollar, MD⁴, Christopher B. Doering, PhD⁵^* and P. Clint Spiegel Jr., PhD¹

¹Chemistry Department, Western Washington University, Bellingham, WA
²Pacific Northwest Center For Cryo-EM, Portland, OR
³National Institute of Biological Standards and Control, Potters Bar, ENG, GBR
⁴Emory University, Children's Center, Atlanta, GA
⁵Aflac Cancer and Blood Disorders Center, Emory University and Children's Healthcare of Atlanta, Atlanta, GA

10:00 AM

429

F5-Atlanta induced Exitron Splicing

Karen L Zimowski, MD¹, Maya Maarouf, PhD¹^*, Julie R Wechsler, BA¹^*, Carlos Lozano¹^*, Seema R Patel, PhD¹, Shannon L Meeks, MD² and Christopher B. Doering, PhD¹^*

¹Aflac Cancer and Blood Disorders Center, Emory University and Children's Healthcare of Atlanta, Atlanta, GA
²Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, GA

10:15 AM

430

A Novel Murine Model Rebalancing Coagulation Enables Gene Therapy Studies for Severe Protein C Deficiency, a Lethal Pro-Thrombotic Disorder

Sarina Levy-Mendelovich, MD¹, Einat Avishai, Msc²^*, Ben Samelson-Jones, MD, PhD³, Rima Dardik, PhD²^*, Tami barazani-Brutman, PhD⁴^*, Yael Nisgav, Msc⁵^*, Tami Livnat⁶^* and Gili Kenet, MD²^*

¹National Hemophilia Center, Sheba Medical Center, Tel Hashomer, Israel; Amalia Biron Research Institute of Thrombosis and Hemostasis, Tel Aviv University, Israel., Tel Aviv, Israel
²National Hemophilia Center, Sheba Medical Center, Tel Hashomer, Israel; Amalia Biron Research Institute of Thrombosis and Hemostasis, Tel Aviv University, Tel Aviv, Israel
³Children's Hospital of Philadelphia, Philadelphia, PA
⁴National Haemophilia Center and Thrombosis & Hemostasis Institute, Sheba Medical Center and Amalia Biron Research Institute of Thrombosis & Hemostasis, Faculty of Medicine, Tel Aviv University, Israel, Ramat Gan, Israel
⁵Rabin Medical Center, Ophthalmology Department, Laboratory of Eye Research Felsenstein Medical Research Center, Petah-Tikva, Israel
⁶National Hemophilia Center, Sheba Medical Center, Tel Hashomer, Israel; Amalia Biron Research Institute of Thrombosis and Hemostasis, Tel Aviv University, Israel., Savyon, Israel

10:30 AM

431

A Novel Cause of Type 1 VWD: Impaired Exocytosis of Weibel-Palade Bodies Due to Biallelic Mutations in Madd

Sophie Hordijk, MSc¹^*, Stijn A. Groten, MSc²^*, Petra E. Bürgisser, MSc³^*, Sebastiaan N.J. Laan, MSc^3,4^*, Georg-Christoph Korenke, MD PhD⁵^*, Tomáš Honzik, MD PhD⁶^*, Diane Beysen, MD PhD⁷^*, Maartje van den Biggelaar, PhD²^*, Tom Carter, PhD⁸^* and Ruben Bierings⁹^*

¹Hematology, Erasmus University Medical Center Rotterdam, Rotterdam, Netherlands
²Molecular Hematology, Sanquin Research, Amsterdam, Netherlands
³Hematology, Erasmus University Medical Center, Rotterdam, Netherlands
⁴Leiden University Medical Center, Leiden, Netherlands
⁵Klinik für Neuropädiatrie und angeborene Stoffwechselerkrankungen, Universitätsklinik für Kinder- und Jugendmedizin, Oldenburg, Germany
⁶Department of Pediatrics and Adolescent Medicine, Charles University and General University Hospital, Prague, Czech Republic
⁷Division of Paediatric Neurology, Department of Paediatrics, Antwerp University Hospital/University of Antwerp, Antwerp, Belgium
⁸Molecular and Clinical Sciences Research Institute, St George's University of London, London, United Kingdom
⁹Hematology, Erasmusmc, University Medical Center Rotterdam, Rotterdam, Netherlands

10:45 AM

432

Neutrophil-Dependent Interferonopathy Promotes Hemophilic Arthropathy

Tomasz Kaminski, PharmD¹, Omika Katoch, PhD²^*, Margaret V. Ragni, MD, MPH^3,4 and Prithu Sundd, PhD^2,5

¹Thrombosis and Hemostasis Program, VERSITI Blood Research Institute, Milwaukee
²Thrombosis and Hemostasis Program, VERSITI Blood Research Institute, Milwaukee, WI
³Hemophilia Center of Western Pennsylvania, Pittsburgh, PA
⁴Department of Medicine and Clinical and Translational Research, University of Pittsburgh, Pittsburgh, PA
⁵Medical College of Wisconsin, Division of Hematology and Oncology, Milwaukee, WI

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^*signifies non-member of ASH

321.O1.6 321. Coagulation and Fibrinolysis: Basic and Translational. A Coagulation Grab Bag