Research Ethics & Policy Series (REPS): Gene Therapy for Sickle Cell Disease: "Research Ethics and Policy Implications" - Alexis A. Thompson, MD, MPH

Gene Therapy for Sickle Cell Disease: Research Ethics and Policy Implications

Alexis A. Thompson, MD, MPH

Professor of Pediatrics (Hematology)
Chief, Division of Hematology
Elias Schwartz MD Endowed Chair in Hematology
Children's Hospital of Philadelphia

Sickle cell disease is an inherited blood disorder that causes severe anemia, and is associated with debilitating pain, chronic organ injury, and early death. Many advances across biomedical research have not translated to improvements in patient outcomes, however, gene therapy as a potential curative approach has renewed interest and hope.

This presentation will summarize accomplishments in sickle cell disease over time, focusing on ethics and policy implications of newer and potentially transformational therapies, acknowledging continued impediments, and exploring opportunities to further make an impact on this condition.

Registration required; Lunch provided
Streaming available via Zoom.