The promise of gene therapy for sickle cell disease

In a clinical trial partly funded by NHLBI, researchers are reporting early success in treatment of sickle cell disease with gene therapy. The findings of the report, published in the New England Journal of Medicine, come from a single patient who has been free of symptoms for over a year. Researchers caution, however, that much more research is needed before gene therapy can become an option for treatment or cure of this condition.