Researchers at the University of California at Los Angeles are working on a gene therapy for bone marrow stem cells that could allow patients with sickle cell disease, who do not have a bone marrow match, to receive transplants using their own modified bone marrow. The treatment involves the addition of an anti-sickling gene in patients' hematopoietic stem cells. Researchers hope to initiate human trials in 2014. Their work is outlined in an online report in the Journal of Clinical Investigation.
Gene therapy for stem cells may improve sickle cell disease treatment
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