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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.

In this Section

Cardiac Therapy
A patient's own stem cells can treat heart failure.

Treatment for Urinary Incontinence
Stem cells are a new twist on an old treatment.

Cancer Stem Cells
A revolution in cancer research is stirring...

Duchenne Muscular Dystrophy
Stem cells may help preserve the strength of boys with this muscle-wasting disease.  

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