Gene therapy offers hope for dogs with muscular dystrophy
Muscular dystrophy (MD) affects approximately 250,000 people in the United States. It also affects pets. Lucky for both populations, there may be a way to curtail it, a new study suggests.
Researchers from the University of Missouri (MU) have successfully treated dogs with the most common form of MD, Duchenne Muscular Dystrophy (DMD). Their findings were published in the National Institute of Health’s PubMed.gov on August 11.
Patients with DMD have a gene mutation that disrupts the production of a protein known as “dystrophin.” Absence of dystrophin starts a chain reaction that eventually leads to muscle cell degeneration and death. Those affected lose their ability to walk and breathe as they get older.
“Due to its size, it is impossible to deliver the entire gene with a gene therapy vector, which is the vehicle that carries the therapeutic gene to the correct site in the body,” said Dongsheng Duan, PhD, one of the study authors.
In the study, the MU team demonstrated for the first time that a common virus can deliver the microgene to all muscles in the body of a diseased dog. The dogs were injected with the virus when they were two to three months old and just starting to show signs of DMD. The dogs are now six to seven months old and continue to develop normally.
“The virus we are using is one of the most common viruses; it is also a virus that produces no symptoms in the human body, making this a safe way to spread the dystrophin gene throughout the body,” Duan said. “These dogs develop DMD naturally in a similar manner as humans. It’s important to treat DMD early before the disease does a lot of damage as this therapy has the greatest impact at the early stages in life.”
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