Gene Therapy for Sickle-Cell Disease?
By Nathan Seppa
By installing a designer gene that offsets the adverse effects of faulty DNA, scientists have prevented sickle-cell disease in mice. Although this potential gene therapy needs much refinement before it can be tested in people, the early success suggests it could lead to a treatment, say the researchers.
Sickle-cell disease is an inherited disorder of red blood cells. It’s caused by a mutation in the gene that encodes beta globin. This protein is a component of hemoglobin, the oxygen-carrying workhorse in red blood cells.