An NHLBI researcher shared encouraging results from a human clinical trial testing a novel gene replacement therapy in people with severe sickle cell disease. The preliminary findings were presented at the 60th Annual Meeting of the American Society of Hematology (ASH), Dec. 1-4, in San Diego.
According to the scientist, the results suggest that the approach has an acceptable level of safety and might help patients consistently produce normal red blood cells instead of the sickle-shaped ones that mark this painful, life-threatening disease.
The experimental treatment involves removing hematopoietic stem cells from the patients’ bone marrow or blood and adding a therapeutic beta globin gene, which is defective in people with sickle cell disease. The cells are then returned to the patients, leading to the production of anti-sickling hemoglobin (T87Q).