Tag: Hematologic disease

The gene editing treatment holds great promise in the fight against sickle cell disease

. . Scientists see promising early results from the first studies testing gene editing for painful, inherited blood disorders that affect millions worldwide, particularly blacks. Doctors hope that the one-time treatment, which permanently changes the DNA in blood cells using a tool called CRISPR, can treat, and potentially cure, sickle cell disease and beta thalassemia. Partial results were presented at a conference of the American Society of Hematology on Saturday and some were published by the New England Journal of Medicine. Doctors described 10 patients who were removed for at least a few months

. . The doctors described 10 patients who are at least a few months away from their treatment, no longer require regular blood transfusions and are free from pain crises.

. . Scientists see promising results from the first studies testing gene editing on sickle cells, which affect many black people.

. . Scientists see promising early results from the first studies testing gene editing for painful, inherited blood disorders that affect millions worldwide, particularly blacks.