UW Medicine: New Gene Therapy Treatment for Duchenne Muscular Dystrophy Shows Promise

UW Medicine-led research has resulted in the discovery of a new gene therapy method for the treatment of Duchenne muscular dystrophy (DMD). The therapy, which focuses on delivering a set of protein packets inside a shuttle vector to replace defective genes within the muscles, shows promise of not only stopping the decline of the muscles affected by this inherited genetic disease but possibly also repairing those muscles in the future. The findings have been published in Nature.
There is no cure for DMD and available treatments and drugs only slow down the disease. DMD affects only males because the gene is on the x chromosome. Patents begin exhibiting symptoms around four years old and usually die in their 20s or 30s.&nbsp;