The Promising Future of Gene Therapy with Duchenne Muscular Dystrophy

The Golden Retriever Muscular Dystrophy (GRMD) dogs were subjected to gene therapy where their faulty genes were replaced with fully functioning ones using a harmless virus, scientifically termed the recombinant adeno-associated virus (rAAV). These dogs also had varying levels of faulty motor skills, and some even moved similarly with normal dogs. With at least a month interval in the treatment period, these dogs were given either a low or high dose of rAAV and jumping and running exercises for observation and comparison. Surprisingly, these dogs still lived for two more years after the trial without any sign of toxicity or immune deficiencies. Clinical studies have also shown that after the long-term microdystrophin gene therapy, their muscle functioning was corrected allowing them to walk, jump, and play like normal dogs.

The result was an exciting development in the study of muscular dystrophy as it suggests that the condition can be treated or is even alterable. It also offers a safe and efficient way to treat DMD. According to professor George Dickson of the Royal Holloway research institute, the study had spectacularly exceeded their expectations. He also noted that it was a tremendous progress for Duchenne MD gene therapy. Although the research utilized the canine model, there is a great possibility that it could also present the same effect in people.