Molecular patches used to treat Duchenne MD

A cocktail of new molecular "patches" have been developed using snippets of DNA-like molecules which can cover mutant DNA sequences that cause Duchenne muscular dystrophy in dogs. And researchers say that the same cocktail approach could work in humans.

Funded in part with money from NIH, the research team used exon-skipping technology to develop the treatment for dogs selected because their version of Duchenne MD is triggered by the same gene as the one in humans. Earlier studies have focused on using this treatment in mice, but researchers wanted to find out if the same strategy could work in larger animals before trying it in humans.

"This is a promising finding," said Duane Alexander, M.D., director of the Eunice Kennedy Shriver National Institute of Child Health and Human Development, one of the NIH institutes that provided partial funding for the study. "It's an important step toward realizing the goal of developing a treatment that could alleviate the symptoms of this disorder."

The study was published on line in the Annals of Neurology and conducted by Toshifumi Yokota, Ph.D., and Eric Hoffman, Ph.D., of Children's National Medical Center, Washington, D.C., and Shin'ichi Takeda, M.D., Ph.D., of the National Center of Neurology and Psychiatry, Ogawa-Higashi, Kodaira, Tokyo, Japan, as well as other researchers at Children's, Carolinas Medical Center, Charlotte, N.C. and the National Center in Japan.

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