A National Institutes of Health (NIH) researcher recently presented preliminary findings of an ongoing multicenter, nationwide clinical trial of gene replacement therapy involving people with severe sickle cell disease. This trial was among several testing experimental sickle cell gene therapies for which preliminary findings were reported at the 60th annual American Society of Hematology meeting in San Diego.
Rubin R. Gene Therapy for Sickle Cell Disease Shows Promise. JAMA. 2019;321(4):334. doi:10.1001/jama.2018.21119
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