FDA Advisers Reviewed a Sickle Cell Treatment

The video discusses sickle cell anemia, highlighting the severe pain and challenges faced by patients. It explains the genetic mutation causing the disease and current treatments like bone marrow transplants. A new gene-editing drug, Exa Cell, is under FDA review, potentially offering a breakthrough treatment. However, concerns about safety and research limitations are noted. The FDA's decision is expected soon, with researchers planning long-term patient monitoring.

What are some of the symptoms described by patients suffering from sickle cell anemia?

How does sickle cell disease affect the shape and function of red blood cells?

Discuss the average life expectancy of patients with sickle cell disease and the factors that contribute to it.

What is the significance of the FDA's decision regarding the new treatment Exa Cell?

What are the potential risks associated with gene editing in the treatment of sickle cell disease?