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First Treatment for Muscular Dystrophy in Sight: Scientists Harness Exon-Skipping in Large Animal to Successfully Treat Duchenne Muscular Dystrophy
Date:3/16/2009

Researchers from Children's National Medical Center and colleagues in Tokyo publish results, video of first successful trial in dogs with Duchenne muscular dystrophy

Washington, DC (PRWEB) March 16, 2009 -- Genetic researchers at Children's National Medical Center and the National Center of Neurology and Psychiatry in Tokyo published the results of the first successful application of "multiple exon-skipping" to curb the devastating effects of Duchenne muscular dystrophy in an animal larger than a mouse. Multiple exon-skipping employs multiple DNA-like molecules as a "DNA band-aids" to skip over the parts of the mutated gene that block the effective creation of proteins.

The study, conducted in Japan and the United States, published this month in the peer-reviewed journal of the American Neurological Association, the Annals of Neurology, treated dogs with naturally occurring canine X-linked muscular dystrophy, a disease which is genetically homologous to the Duchenne muscular dystrophy that strikes 1 of every 3,500 boys born in the United States and worldwide each year.

Duchenne muscular dystrophy, one of the most common lethal genetic disorders, is an X-linked genetic mutation that causes an inability of the body's c
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First Treatment for Muscular Dystrophy in Sight: Scientists Harness Exon-Skipping in Large Animal to Successfully Treat Duchenne Muscular Dystrophy 
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