Stem cells taken from Duchenne muscular dystrophy patients and genetically modified eased symptoms of the disease in mice, strengthening their muscles and allowing them to run longer on a treadmill, researchers reported Wednesday in the journal Cell Stem Cell.
The patients have a nonfunctional form of a protein called dystrophin. Researchers modified the muscle cells from the patients to produce a shortened but still effective form of dystrophin. Injected into the mice's bloodstreams, the cells migrated to the site of damaged muscles and were incorporated into muscle tissues.
