Gene Therapy Gives Clue to Treatment

CHAPEL HILL, N.C., Oct. 6 (UPI) — A first clinical trial to replace a genetic defect in patients with muscular dystrophy has yielded an unexpected and encouraging result, U.S. researchers say.

University of North Carolina at Chapel Hill School of Medicine researchers have made a discovery about a muscle protein called dystrophin, an essential protein that is deficient in those suffering the disease, a university release said.

The illness occurs when a gene on the X chromosome fails to make the vital protein. In the gene therapy trial it was found that the immune system of muscular dystrophy patients, once thought to be devoid of dystrophin, is “primed” by the prior existence of a tiny amount of the vital protein that escaped the effects of the mutation.

The finding suggests some patients may benefit from immunosuppression prior to receiving gene therapy.

“We can now use this new information to adapt our approach to make gene therapy more likely to succeed,” Dr. R. Jude Samulski, professor of pharmacology and director of the Gene Therapy Center at UNC, said. “Right now we are searching for a way to cure this disease, not just care for it, but truly cure it. So we realize that this effort is going to be an iterative process, with the accumulation of a number of lessons along the way to help us succeed.”

The study was published in the New England Journal of Medicine.

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