Gene therapies show promise for sickle cell disease

By Sheilah Kast,

Published June 14, 2024 at 9:55 AM EDT

Baltimore middle school student Cimone Vaughan lives with sickle cell disease. Her mother, Nikia Vaughan, is the executive director of the Maryland Sickle Cell Disease Association. Credit: Nikia Vaughan — Baltimore middle school student Cimone Vaughan lives with sickle cell disease. Her mother, Nikia Vaughan, is the executive director of the Maryland Sickle Cell Disease Association. *Credit: Nikia Vaughan*

June 19th is World Sickle Cell Awareness Day. This painful rare disease affects an estimated 100,000 people in the United States. It’s more prevalent among people of African ancestry. About 1 in 13 Black or African American babies are born with sickle cell trait, which could be passed to their children.

What promise do new cell-based gene therapies hold?

We speak with Cimone Vaughan, a 12-year-old living with sickle cell disease, and her mother, Nikia Vaughan, executive director of the Maryland Sickle Cell Disease Association.

Then, Dr. Jason Fixler, a doctor of pediatric hematology oncology at Lifebridge Health breaks down misconceptions about the disease.

Links:

CDC Sickle Cell Disease
Sickle Cell Improvement in the Northeast Region through education project
FDA approves two sickle cell therapies, including first CRISPR medicine
Baltimore is a city heavily impacted by Sickle Cell Disease as awareness day approaches