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Duchenne Muscular Dystrophy Research at the McGowan Institute

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.

Contact Us

McGowan Institute for Regenerative Medicine
450 Technology Drive
Suite 300
Pittsburgh, PA 15219

Read information on campus shuttles.

Phone: 412-624-5500
Fax: 412-624-5363
Email: McGowan@pitt.edu

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