Gene therapy for stem cells may improve sickle cell disease treatment

07/3/2013 | Los Angeles Wave

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.

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