National Academy of Sciences Committee on Human Gene Editing Trevor Thompson, ABD CE0 – Sickle Cell Foundation of Tennessee

What is Sickle Cell Disease? Sickle Cell Disease is an inherited blood disorder that affects red blood cells. People with sickle cell disease have red blood cells that contain mostly hemoglobin S, an abnormal type of hemoglobin. Sometimes these red blood cells become sickle-shaped (crescent shaped) and have difficulty passing through small blood vessels. Treatment of complications often includes antibiotics, pain management, intravenous fluids, blood transfusion, and surgery, all backed by psychosocial support, bone marrow transplant. There is currently no universal cure for Sickle Cell Disease.

Data Approximately 70,000 to 100,000 people affected with Sickle Cell Disease (SCD) -USA 1 in 500 African American 1 in 1200 Hispanics Estimated 2.5 million with Sickle Cell Trait (SCT) –USA Millions Affected Nationally

Let’s Have A Conversation 1. What public process has occurred in which the larger patient/affected community has been a participant – are there examples of optimal processes and why? What kinds of conversations around gene editing have affected communities been involved in? 2. How can policy makers be sure that they are hearing from a fully representative point of view from affected patients? Who is representative of these different patient/affected communities? 3. Who should speak for patients not yet born or not yet affected by a particular disease or condition? 4. For the individual speakers, what is you opinion or perspective on the use of gene editing and what is your view based on?