Dr. Donald Kohn and colleagues at the UCLA Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research successfully established the foundation for a gene therapy approach to treat sickle cell disease (SCD). Using patients own hematopoietic (blood producing) stem cells, Kohn introduced an anti-sickling gene into the stem cells to capitalize on their self-renewing potential that will create for the patients a continual source of healthy red blood cells that do not sickle. The breakthrough technique to treat sickle cell disease is scheduled to begin clinical trials by early 2014. The study was published online ahead of press today in the Journal of Clinical Investigation.

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