Conference to focus on preventing and reversing consequences of sickle cell disease

Leading experts in the field of sickle cell disease (SCD) research will convene in Washington, D.C., for the Physiological and Pathophysiological Consequences of Sickle Cell Disease conference (November 6–8). The conference, organized by the American Physiological Society (APS), will explore SCD-;the world's most prevalent single-gene mutation disease-; and new research on preventing and reversing its deadly consequences.

SCD affects millions of people around the world and more than 100,000 people in the U.S. In recent years, the prognosis of people with SCD has improved, but the only way to cure SCD is through a risky bone marrow transplant. "Currently, there is only one FDA-approved treatment for the disease," said conference chair Dexter L. Lee, PhD, of Howard University in Washington, D.C. "Researchers across the country and around the globe are making progress in our understanding of the disease and the discovery of new therapies to serve the SCD community." The goal of the conference is to convene an internationally recognized, interdisciplinary group of investigators to present and discuss current basic and clinical research findings in SCD and to develop new ideas and directions for future research in SCD.

Much of the science presented at the meeting will focus on translational research and new potential therapies, including causes of and treatment for pain, clotting and inflammation, and cell and gene therapy. The abstract-driven program will highlight both established researchers and trainees and early-career investigators giving oral and poster presentations of their work.

Program Highlights

Monday, November 6, 2017

Opening Remarks: Harvey Luksenburg, National Institutes of Health; National, Heart, Lung and Blood Institute

Tuesday, November 7, 2017

Plenary Lecture Speaker: Wayne A. I. Frederick, Howard University, Washington, D.C.

Symposia I: Neural Circuits and Neurovascular Physiology Chair: Thomas D. Coates, University of Southern California

Targeting Pain at its Source in Sickle Cell Disease Speaker: Kalpna Gupta, University of Minnesota

Symposia II: SCD Gene Therapy, Gene Editing, and Pharmacological Treatment Chair: David Williams, Harvard Stem Cell Institute, Cambridge, Mass.

Gene Therapy for Hemoglobinopathies: The Challenge to Find a Cure Speaker: Giuliana Ferrari, San Raffaele Telethon Institute for Gene Therapy, Milan, Italy

Discovery of Pharmacologic Fetal Hemoglobin Inducing Agents for Sickle Cell Disease Speaker: Betty Pace, Augusta University, Georgia

Symposia III: Cell Therapy Chair: Betty Pace, Augusta University, Georgia

Control of HbF Silencing: Implications for Genetic and Pharmacologic Induction of HbF for Therapy Speaker: Stuart Orkin, Harvard University, Cambridge, Mass.

CRISPR/Cas9 Enhanced Sickle Gene Correction in Human and Mouse Hematopoietic Stem Cells Speaker: Tim Townes, University of Alabama at Birmingham

Symposia IV: Small Molecules to Treat SCD Chair: James Douglas Engel, University of Michigan

KEAP1-NRF2 Antioxidant Response System and Sickle Cell Anemia Speaker: Masayuki Yamamoto, Tohoku University, Japan

Oral Tetrahydrouridine and Decitabine for Non-cytotoxic Epigenetic Modification of Sickle Cell Disease: A Randomized Phase 1/2 Study Speaker: Yogen Saunthurarajah, Cleveland Clinic

RN-1, an LSD-1 Inhibitor, Induces HbF in the Baboon (P. anubis) and Reduces Mitochondria-containing RBC in a SCD Mouse Model Speaker: Angela Rivers, University of Illinois, Chicago

Wednesday, November 8, 2017

Symposia V: Renal and Vascular Physiology Chair: Jennifer Pollock, University of Alabama at Birmingham

Sickle Cell Disease: When Endothelin Becomes a Nephrotoxic and Proinflammatory Cytokine Speaker: Pierre-Louis Tharaux, INSERM U970, Paris, France

Symposia VI: Lung Physiology and Pathophysiology Chair: Steffen Meiler, Augusta University, Georgia

TBD Speaker: Solomon Ofori-Acquah, University of Pittsburgh

Symposia VII: Red Cell Physiology Chair: Sergei Nekhai, Howard University, Washington, D.C.

Developmental Regulation of Erythroid Self-renewal Speaker: James Palis, University of Rochester Medical Center

Pathobiology of Sickle Red Cells: Implications for Pathophysiology of Sickle Cell Disease Speaker: Mohandas Narla, New York Blood Center

Symposia VIII: Coagulation and Thrombosis Chair: Felicity N. Gavins, Louisiana State University Health Sciences Center, and Rafal Pawlinski, University of North Carolina

De-clotting Sickle Cell Disease Speaker: Rafal Pawlinski, University of North Carolina

Promoting the Resolution of Inflammation in Sickle Cell Disease Speaker: Felicity N. Gavins, Louisiana State University Health Sciences Center