Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a genetic disorder afflicting one in 3,500 boys, most of whom will die in adolescence or young adulthood as their muscles — including their hearts — weaken. The problem is that their muscle cells don't produce the essential protein dystrophin.
McGowan Institute researchers have identified and isolated a line of stem cells in muscles that offers new hope for Duchenne patients. When these stem cells are genetically engineered to contain a gene encoding for dystrophin, they produce dystrophin in Duchenne-affected muscles. This work may produce a minimally invasive treatment in which the patient's own stem cells are isolated, engineered in the lab to carry a functioning dystrophin gene, then reinjected into muscle.