Researchers Develop Promising Method to Treat Sickle Cell Disease

March 9, 2015

(Bioscience Technology) – UCLA stem cell researchers have shown that a novel stem cell gene therapy method could lead to a one-time, lasting treatment for sickle cell disease — the nation’s most common inherited blood disorder. Published March 2 in the journal Blood, the study led by Dr. Donald Kohn of the UCLA Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research outlines a method that corrects the mutated gene that causes sickle cell disease and shows, for the first time, that the gene correction method leads to the production of normal red blood cells.