Home United States USA — Science Gene editing treatment shows promise in sickle cell disease fight

Gene editing treatment shows promise in sickle cell disease fight

236
0
SHARE

Scientists are seeing promising early results from the first studies to examine the effects of gene editing on painful, inherited blood disorders that plague millions worldwide, especially black people.
Scientists are seeing promising early results from the first studies to examine the effects of gene editing on painful, inherited blood disorders that plague millions worldwide, especially black people. 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 at an American Society of Haematology 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 that had plagued their lives before. Victoria Gray, the first patient in the sickle cell study, was a lifelong sufferer of severe pain bouts that often sent her to hospital. “I had aching pains, sharp pains, burning pains, you name it. That’s all I’ve known my entire life,” said Ms Gray,35, who lives in Forest, Mississippi. “I was hurting everywhere my blood flowed.” Since her treatment a year ago, Ms Gray has weaned herself from pain medication 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 for now is a bone marrow transplant from a closely matched donor without the disease, such as a sibling.

Continue reading...