Gene treatment shows promise for Duchenne muscular dystrophy

Mar 28, 2009
By staff
Washington -- A research study funded by the National Institutes of Health reveals a new treatment for the canine form of muscular dystrophy that could help treat the human form of the disease.

Published online in the Annals of Neurology, the study found that, using a genetic technology called "exon skipping," scientists could use pieces of DNA-like molecules and apply them as patches to cover up mutant DNA sequences. The dog's body can then make a functional version of the muscle protein affected by Duchenne muscular dystrophy.

The same gene is affected in the human form of the disease.

The study was conducted by Dr. Toshifumi Yokota and Dr. Eric Hoffman of Children's National Medical Center in Washington, D.C., and Dr. Shin'ichi Takeda of the National Center of Neurology and Psychology in Tokyo, as well as researchers at Children's, Carolinas Medical Center in Charlotte, N.C., and the National Center in Japan.