Sickle Cell Disease

It is no more than a reversal of an A and T base pair in a point on Chromosome 11 that results in a substitution of valine for glutamic acid that causes sickle cell anemia. To correct that mutation, researchers in the Johns Hopkins cell engineering lab used the CRISPR gene editing technique to “heal” the defect in a sufferer’s induced pluripotent stem cells and then, outside the body, grow up a hematopoietic cure. MORE

Image Credit: University of Utah