She is the first sickle cell patient to receive a revolutionary gene therapy using a tool called CRISPR. She now shares her symptom-free success story on a global stage.
Sickle cell disease is an inherited red blood cell disorder that affects the oxygen-carrying proteins in red blood cells, contorting them into a crescent moon shape, making movement through the body and oxygen delivery to tissues difficult. About 100,000 Americans have it, but it's most prevalent in individuals with African ancestry, occurring in 1 out of every 365 births.
CRISPR is a tool that makes precise cuts in DNA at a specific location. In this case, the cuts increase the production of fetal hemoglobin. Frangoul and a team at Sarah Cannon Research Institute and HCA Healthcare's The Children's Hospital At TriStar Centennial in Nashville collected Gray's stem cells, a lab reengineered them, and then he transplanted them back into Gray's bloodstream."I would pinch my face and thigh, thinking my body was numb, or something was wrong, because I wasn't in pain," she says."But then I realized, no — it's just working.
CRISPR has been tested in at least 75 people since Gray's treatment and could be approved in the United States this year.
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