Gene Therapy for Sickle-Cell Disease?

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.

Three months after a transplant of bone marrow predisposed to sickle-cell disease, a mouse produced deformed red blood cells (left).