NPR

Gene-Edited 'Supercells' Make Progress In Fight Against Sickle Cell Disease

Researchers edited the DNA in bone marrow cells taken from a Mississippi woman with sickle cell disease to produce a treatment that could alleviate the excruciating effects of her inherited illness.
As part of a clinical trial to treat sickle cell disease, Victoria Gray (center) has vials of blood drawn by nurses Bonnie Carroll (left) and Kayla Jordan at TriStar Centennial Medical Center in Nashville, Tenn. Source: Meredith Rizzo/NPR

Doctors are reporting the first evidence that genetically edited cells could offer a safe way to treat sickle cell disease, a devastating, incurable disorder that afflicts millions of people around the world.

Billions of cells that were genetically modified with the powerful gene-editing technique called CRISPR have started working, as doctors had hoped, inside the body of the first sickle cell patient to receive the experimental treatment, according to highly anticipated data released Tuesday.

The edited cells are producing a crucial protein at levels that have already exceeded what doctors thought would be needed to alleviate the excruciating, life-threatening complications of the genetic blood disorder, the early data show. Moreover, the cells appear to have already started to spare the patient from the agonizing attacks of pain that are the hallmark of the disorder.

"We are very, very excited," says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating the patient. "This preliminary data shows for the first time that gene editing has actually helped a patient with sickle cell disease. This is definitely a huge deal."

Frangoul and other researchers caution, however, that the results involve just one patient who was only recently treated. It is far too soon to answer the most crucial questions: Will the modified-cell treatment continue to improve the patient's

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