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NIH researcher presents encouraging results for gene therapy for severe sickle cell disease

from NIH

A scientist from the National Institutes of Health will present promising, early results from a human clinical trial testing a novel gene replacement therapy in people with severe sickle cell disease. Preliminary findings 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. more


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