Public Health Webinar Series on Blood Disorders
CDC’s Division of Blood Disorders and Public Health Genomics (DBDPHG) is proud to offer its Public Health Webinar Series on Blood Disorders. The purpose of this series is to provide evidence-based information on new research, interventions, emerging issues of interest in blood disorders, as well as innovative approaches in collaborations and partnerships. We invite you to join us in this series.
June 22, 2023
Stefano Rivella, PhD
Professor of Pediatrics, Children’s Hospital of Philadelphia
Scientific Director of The Sickle Cell and Red Cell Disorders Curative Therapy Center,
Children’s Hospital of Philadelphia
Sujit Sheth, MD
Chief, Division of Pediatric Hematology/Oncology
Professor of Clinical Pediatrics, Weill Cornell Medicine
Muin J. Khoury MD, PhD
Director, Office of Genomics and Precision Public Health,
National Center on Birth Defects and Developmental Disabilities,
Centers for Disease Control and Prevention
Although relatively new in terms of clinical application, several gene therapy-based treatments have, in recent years, received approval from the Food and Drug Administration (FDA) and begun to be used in real world settings in the United States. In addition, clinical trials using either gene transfer or genome editing continue to show promise, with the potential to impact treatment for patients with a wide range of hereditary disorders in the future. This webinar will showcase thalassemia as a case example in emerging approaches in gene therapy.
In this presentation, Drs. Rivella and Sheth will review the status of the FDA-approved gene therapy process for thalassemia, discussing the science behind it, the results of the clinical trials associated with it, and the clinical implications as gene therapy begins to be applied in a real-world setting. In addition, they will explore future clinical trials involving potential alternative gene therapy treatments.
To view webinars by year or disorder, select “Year” and “Disorder” area below.