Surgery prof makes advance in muscular dystrophy fight

When Hansell Stedman was growing up, his brother's debilitating hereditary illness -- muscular dystrophy -- seemed so far from a cure that Stedman never believed he would research it. Now, as this April's cover of Nature Medicine attests, the 40-year-old Penn surgery professor may be on his way to finding it. Stedman -- the principal author of the study published in the prestigious scientific journal -- and his team were able to restore a group of critical proteins to muscle tissue that had lost these proteins from the onslaught of a muscular dystrophy. The loss of these proteins typically results in muscle weakness that can lead to paralysis and even death. Experts in the field are calling Stedman's breakthrough study very encouraging. "These exciting achievements confirm that we're on the right track," Donald Wood, the Muscular Dystrophy Association's director of science technology, said in a press release. "We're more committed than ever to begin clinical trials of gene therapy for muscular dystrophy." Muscular dystrophy is actually a group of diseases, all hereditary, characterized by the weakening of the body's muscles. It is usually fatal, according to the MDA World Wide Web site. The different types vary in the age of onset and the rate of progression. Stedman, who works at Penn's Institute for Human Gene Therapy, used gene therapy techniques to help repair the muscle cells of hamsters ravaged by a muscular dystrophy called Limb Girdle. When Stedman and team member James Greelish, a surgical resident at the Hospital of the University of Pennsylvania, delivered certain genes to the muscle cells, they found that those cells were able to produce key proteins in large quantities -- raising the hope that the restoration of full muscle function could some day be possible. Leonard Su, a medical student at HUP, cloned the genes that would be effective in producing the desired proteins, but Stedman still needed a way to transport those genes to the muscles. James Wilson, director of the Institute for Human Gene Therapy, had done extensive work with a harmless virus that seemed like the perfect carrier for Stedman's genes. But the virus was too big to exit the main vessels of the circulatory system and reach the targeted muscle cells. To solve this quandary, Stedman used a drug to enlarge the openings connecting main blood vessels to smaller branches, enabling the virus to escape the vessel and get to the damaged cells. While Stedman treated Limb Girdle muscular dystrophy, his technique may also work with a type called Duchenne muscular dystrophy, a fatal disease found in one of every 3,000 men, according to Stedman. "Duchenne muscular dystrophy may well be the most common lethal single-gene defect in man," Stedman said. The next step is to prove the new therapy is safe and effective in humans, a goal Stedman said he hopes to realize this year.

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