OC 04 - Hemophilia Basic Science - FVIII and FIX

Moderator(s)

• 

  Steven Pipe, MD

  Professor of Pediatrics and Pathology
  Pediatrics and Pathology
  University of Michigan, Ann Arbor, MI, USA
  Ann Arbor, Michigan, United States
• 

  Orla Rawley, PhD (she/her/hers)

  Post-Doctoral Fellow
  Pathology and Molecular Medicine
  Queen's University, Pathology and Molecular Medicine
  Kingston, Ontario, Canada

Presentations:

• 13:00 – 13:15 ET

  Exploring the hemostatic properties of extravascular factor IX in a hemophilia B mouse model
  

  Presenting Author: Bonnie Chu (she/her/hers) – McMaster University

  Co-Author: Wajeeha Zaheer (she/her/hers) – McMaster University

  Co-Author: Paul Tieu – Thrombosis & Atherosclerosis Research Institute, McMaster University, Hamilton, Canada

  Co-Author: Hasam Madarati – McMaster University

  Co-Author: Colin Kretz, PhD – McMaster University

  Co-Author: Peter L. Gross, MD MSc FRCP(c) (he/him/his) – Department of Medicine, McMaster University

  Co-Author: Anthony KC. Chan, MBBS – McMaster University, McMaster Children’s Hospital, Hamilton, Canada

  Co-Author: Davide Matino, MD (he/him/his) – Department of Medicine, Division of Hematology, McMaster University, Hamilton, ON
• 13:15 – 13:30 ET

  B-Domain deleted Factor VIII shows clear differences in intracellular processing and cellular responses when compared to full length Factor VIII
  

  Presenting Author: Rawya Al Rifai – Institute of Experimental Haematology and Transfusion Medicine and Center for Rare Diseases, University Hospital Bonn, Bonn, Germany

  Co-Author: Muhammad Ahmer Jamil – Institute of Experimental Haematology and Transfusion Medicine and Center for Rare Diseases, University Hospital Bonn, Bonn, Germany

  Co-Author: Salime El Hazzouri – University Clinic Bonn, Institute of experimental Haematology and Transfusion medicine

  Co-Author: Mira Ibrahim, MS – Institute of Experimental Haematology and Transfusion Medicine and Center for Rare Diseases, University Hospital Bonn, Bonn, Germany

  Co-Author: Nicole Nüsgen – Institute of Experimental Haematology and Transfusion Medicine and Center for Rare Diseases, University Hospital Bonn, Bonn, Germany

  Co-Author: Johannes Oldenburg, MD (he/him/his) – University Clinic Bonn AöR, Institute of Experimental Haematology and Transfusion Medicine, Bonn, Germany

  Co-Author: Osman EL-Maarri, Priv Doz, PhD (he/him/his) – Institute of Experimental Haematology and Transfusion Medicine and Center for Rare Diseases, University Hospital Bonn, Bonn, Germany
• 13:30 – 13:45 ET

  Prospective FVIII intracellular trafficking routes involving GABARAP
  

  Presenting Author: Salime El Hazzouri – University Clinic Bonn, Institute of experimental Haematology and Transfusion medicine

  Co-Author: Nicole Nüsgen – Institute of Experimental Haematology and Transfusion Medicine and Center for Rare Diseases, University Hospital Bonn, Bonn, Germany

  Co-Author: Rawya Al Rifai – Institute of Experimental Haematology and Transfusion Medicine and Center for Rare Diseases, University Hospital Bonn, Bonn, Germany

  Co-Author: Heike Singer – University Clinic Bonn, Institue of experimental Haematology and Transfusion medicine

  Co-Author: Nasim Shahidi Hamedani, PharmD PhD (she/her/hers) – University Hospital Bonn; Institute of Experimental Haematologie and Transfusion Medicine

  Co-Author: Johannes Oldenburg, MD (he/him/his) – University Clinic Bonn AöR, Institute of Experimental Haematology and Transfusion Medicine, Bonn, Germany

  Co-Author: Osman EL-Maarri, Priv Doz, PhD (he/him/his) – Institute of Experimental Haematology and Transfusion Medicine and Center for Rare Diseases, University Hospital Bonn, Bonn, Germany
• 13:45 – 14:00 ET

  A novel tailored correction approach for recurrent hemophilia-causing nonsense mutations through AntiCodon-Engineered suppressor tRNAs
  

  Presenting Author: Maria Francesca Testa, PhD – Department of Life Sciences and Biotechnology and LTTA Center, University of Ferrara, Ferrara 44121, Italy

  Co-Author: Jhon D. Lueck – Department of Physiology and Pharmacology, University of Rochester School of Medicine and Dentistry, Rochester, NY 14642, USA

  Co-Author: Francesco Bernardi – Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, Ferrara (Italy)

  Co-Author: Mirko Pinotti – Department of Life Sciences and Biotechnology and LTTA Center, University of Ferrara, Ferrara 44121, Italy

  Co-Author: Alessio Branchini – Department of Life Sciences and Biotechnology and LTTA Center, University of Ferrara, Ferrara 44121, Italy
• 14:00 – 14:15 ET

  Structural, functional, and immunogenicity implications of F9 gene recoding
  

  Presenting Author: Upendra K. Katneni, PhD – US Food and Drug Administration

  Co-Author: Aikaterini Alexaki – US Food and Drug Admininstration

  Co-Author: Ryan Hunt – US Food and Drug Administration

  Co-Author: Nobuko Hamasaki-Katagiri – US Food and Drug Administration

  Co-Author: Gaya Hettiarachchi – US Food and Drug Administration

  Co-Author: Brian Lin, PhD – US Food and Drug Administration

  Co-Author: Leonid Parunov, PhD (he/him/his) – US Food and Drug Administration

  Co-Author: Mikhail V. Ovanesov – US Food and Drug Administration

  Co-Author: Darón I. Freedberg – US Food and Drug Administration

  Co-Author: Haim Bar – University of Connecticut

  Co-Author: Anton A. Komar – Cleveland State University

  Co-Author: Zuben E. Sauna – U.S. Food and Drug Administration

  Co-Author: Chava Kimchi-Sarfaty – US Food and Drug Administration