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Researchers develop DNA 'patch' for canine form of muscular dystrophy
Date:3/16/2009

mutations that affect multiple exons, and so would require more than one kind of patch. The dogs in the study carried a mutation affecting multiple exons. The researchers used a cocktail containing multiple DNA patches to bypass the affected exons.

The patches, DNA-like molecules called morpholinos, are manufactured in a laboratory. Injections of the morpholino cocktail directly into the dogs' bloodstream curbed deterioration of the animals' skeletal muscles and improved muscle functioning. The injections resulted in widespread production of dystrophin-like protein, at about 26 percent of normal levels. However, the treatment was unable to prevent deterioration of the animals' hearts. The researchers theorized that the muscles of the heart are less porous than the skeletal muscles, and did not absorb sufficient quantities of the morpholinos to curb the deterioration.

The researchers said that other means of delivering the morpholinos to the heart would need to be explored.

They added, however, that the study results showed that it would be possible to use a cocktail of morpholinos to patch the multiple mutations that occur in the human form of the disorder.


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Contact: Robert Bock
bockr@mail.nih.gov
301-496-5133
NIH/National Institute of Child Health and Human Development
Source:Eurekalert

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