Gene-editing treatment shows promise for sickle cell disease

Scientists are seeing promising early results from the first studies testing gene editing for painful, inherited blood disorders that plague millions worldwide.

Doctors hope the one-time treatment, which involves permanently altering DNA in blood cells with a tool called CRISPR, may treat and possibly cure sickle cell disease and beta thalassemia.

Partial results were presented Saturday at an American Society of Hematology conference and some were published by the New England Journal of Medicine.

Doctors described 10 patients who are at least several months removed from their treatment. All no longer need regular blood transfusions and are free from pain crises that plagued their lives before.

Victoria Gray, the first patient in the sickle cell study, had long suffered severe pain bouts that often sent her to the hospital.

“I had aching pains, sharp pains, burning pains, you name it. That’s all I’ve known my entire life,” said Gray, 35, who lives in Forest, Mississippi. “I was hurting everywhere my blood flowed.”

Since her treatment a year ago, Gray has weaned herself from pain medications she depended on to manage her symptoms.

“It’s something I prayed for my whole life,” she said. “I pray everyone has the same results I did.”

Sickle cell affects millions, mostly Black people. Beta thalassemia strikes about one in 100,000 people. The only cure now is a bone marrow transplant from a closely matched donor without the disease like a sibling, which most people don't have.

Both diseases involve mutations in a gene for hemoglobin, the substance in red blood cells that carries oxygen throughout the body.

In sickle cell, defective hemoglobin leads to deformed, crescent-shaped blood cells that don’t carry oxygen well. They can stick together and clog...