U.S, Japanese Scientists Test Treatmentfor Muscular Dystrophy: Pilot StudyShows "Exon Skipping" DNA Therapy Effective in Dogs
Washington — Scientists from the United States and Japan successfully tested a treatment for Duchenne muscular dystrophy in dogs, paving the way for a possible treatment of the disease in humans.
The treatment allows cells to skip over lethal mutations in DNA and produce functional proteins.
Such “exon-skipping” therapy has never before been tested in an animal larger than a mouse. The results were published online March 13 in the journal Annals of Neurology.
"This trial makes the much-talked-about promise of exon skipping as a systemic treatment for Duchenne muscular dystrophy in humans a real possibility in the near term," said Toshifumi Yokota, lead author of the study.
“These findings demonstrate that exon skipping is a very real and promising treatment strategy for Duchenne muscular dystrophy,” said Sharon Hesterlee, senior vice president of the Muscular Dystrophy Association, a nonprofit organization that funds related research.
Muscular dystrophies are a collection of more than 30 genetic diseases that cause progressive weakness of the skeletal muscles that control movement. The diseases vary in severity. Some, such as Becker muscular dystrophy, have mild symptoms, caused by mutations that produce a shortened form of the dystrophin protein.
Duchenne muscular dystrophy, the most common form, affecting approximately one in 3,500 males worldwide, is caused by mutations in the dystrophin gene that prevent the production of dystrophin protein. Symptoms begin between 3 years and 5 years of age and progress rapidly. Teenagers often cannot walk and require a respirator to breathe. Duchenne is fatal and, like all muscular dystrophies, there currently is no treatment.
The new study used beagles with a mutation in the canine dystrophin gene.
“Many efforts have focused on treating dogs with muscular dystrophy, as it is widely expected that what works in the dogs will work in humans," said Eric Hoffman, professor of pediatrics at Children's National Medical Center and a senior author of the study.
Reprinted with the permission of the Department of State.
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