Dr. Donald Kohn in the lab with Megan Poto

Dr. Donald Kohn and colleagues show that a novel stem cell gene therapy method could one day provide a one-time, lasting treatment for the most common inherited blood disorder in the United States – sickle cell disease.

The stem cell research published today in the journal Blood, outlines a method that corrects the mutated gene that causes sickle cell disease and shows, for the first time, the gene correction method leads to the production of normal red blood cells that do not sickle. The fascinating gene correction technique used specially engineered enzymes, called zinc-finger nucleases, to ‘cut’ out the mutated genetic code and replace it with a corrected version that repairs the beta-globin mutation.

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