According to a new study published on March 2 in the journal Blood, researchers from the University of California, Los Angeles 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.
The study led by Dr Donald Kohn, researcher at the Eli & Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, 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.
The stem cell gene therapy method described in the study seeks to correct the mutation in the beta-globin gene so bone marrow stem cells produce normal, circular-shaped blood cells. The technique used specially engineered enzymes, called zinc-finger nucleases, to eliminate the mutated genetic code and replace it with a corrected version that repairs the beta-globin mutation.
The research showed that the method holds the potential to permanently treat the disease if a higher level of correction is achieved. The results suggest the future direction for treating genetic diseases will be by correcting the specific mutation in a patient’s genetic code.