The journal Science published the first clear evidence that reprogrammed stem cells can correct an inherited disease.
Experiments in mice may lead to new treatments for the disease, study suggests.
Experts discuss how to cure a chronic disease
In theory, it’s a simple idea: Overcome a defective gene by plugging in a healthy one.
In February 2011 the U.S. Food and Drug Administration approved Corifact, the first product intended to prevent bleeding in people with the rare genetic defect congenital Factor XIII deficiency.