Posted on January 7, 2022

First Sickle Cell Patient Treated With CRISPR Gene-Editing Still Thriving

Rob Stein, NPR, December 31, 2021

For more than a year, Victoria Gray’s life had been transformed. Gone were the sudden attacks of horrible pain that had tortured her all her life. Gone was the devastating fatigue that had left her helpless to care for herself or her kids. Gone were the nightmarish nights in the emergency room getting blood transfusions and powerful pain medication.

But one big question remained: Would the experimental treatment she got to genetically modify her blood cells keep working, and leave her free from the complications of sickle cell disease that had plagued her since she was a baby?

More than another year later, the answer appears to be: Yes.

“I’m doing great,” Gray, now 36, said during a recent interview from her home in Forest, Miss. with NPR, which has had exclusive access to chronicle her experience for more than two years.


Victoria’s so traumatized by a life of sickle cell that just getting a cold still terrifies her. A simple cold had been one of many things that could trigger a terrible attack of the painful symptoms of the disease.


She’s doing so well for so long that she’s officially no longer in the landmark study she volunteered for. That involved doctors taking cells out of her bone marrow, and editing a gene in the cells in their lab, using the revolutionary gene-editing technique known as CRISPR. CRISPR allows scientists to make very precise changes in DNA much more easily than ever before. Many think it will revolutionize medicine.


And it appears to have worked, for Gray and other patients. Doctors have now treated at least 45 patients with sickle cell and a related condition known as beta thalassemia, and reported data indicating it’s working for at least 22 of them.