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First treatment for muscular dystrophy in sight: Scientists successfully harness exon-skipping
Date:3/16/2009

WASHINGTON, DCGenetic 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 cells to effectively create dystrophinwhich builds muscle tissue. "Exon-skipping" employs synthetic DNA-like molecules called antisense as a DNA band-aid to skip over the parts of the gene that block the effective creation of dystrophin. Because the gene's mutation could affect any of its 79 exons and sometimes more than one single exon at a time, scientists employed a "cocktail" of antisense called morpholinos to extend the range of this application. By skipping more than a single exon, this so-called DNA band-aid becomes applicable to between 80 and 90 percent of Duchenne muscular dystrophy patients, including the mutation foun
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Contact: Jennifer Leischer
jleische@cnmc.org
202-476-4500
Children's National Medical Center
Source:Eurekalert

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