Gene-editing treatment shows promise for sickle cell disease
December 6, 2020
Scientists are seeing promising early results from the first studies testing gene editing for 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 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...