(March 16, 2009 - Insidermedicine) The first potential treatment for Duchenne muscular dystrophy has been successfully used in dogs, according to research in the Annals of Neurology.

Here is some information about Duchenne muscular dystrophy:

•    It is a genetic disorder of the X chromosome mainly affecting boys.

•    It is caused by any of several mutations on a gene that result in the body being unable to properly produce dystrophin, which is needed to build muscle tissue.

•    Duchenne causes muscle weakness and wasting. Its effects on the heart and breathing muscles generally result in death by the early 30s.

Researchers from Children’s National Medical Center and the National Center of Neurology and Psychiatry in Tokyo developed a cocktail of molecules, called morpholinos, that affect how a gene is “read” by the body. They tell the body to skip over the mutated portion of the gene.

Using the cocktail in dogs with a canine form of muscular dystrophy genetically identical to Duchenne did result in the preservation of muscle strength. The heart muscle, however, was less well protected than the other muscles of the body.

We had a chance to speak with Dr. Eric Hoffman, a senior author on this study, who offered some further insight.

Today’s research demonstrates a new approach to treating genetic conditions, in which defective parts of a gene are simply skipped over.

For Insidermedicine in Depth, I'm Dr. Kelly Schweitzer.