Gene Therapy Promising For Neuromuscular Disease Treatment In Dogs And

When our dogs develop debilitating and life-ending disorders, our hearts break. Researchers from the University of Washington believe they have promising treatment options for dogs who have a previously incurable neuromuscular disease.

Researchers from the University of Washington, in collaboration with Harvard University, Virginia Tech, The Medical College of Wisconsin, INSERM (French National Institute of Health and Medical Research), and Genethon are encouraged by their latest findings about the restoration of muscle strength and the prolongation of life expectancy for dogs who have a rare inherited neuromuscular disorder called myotubular myopathy, or MTM.

MTM is a disease caused by a gene mutation in male dogs. In affected dogs, those genes would typically make a type of protein called myotubularin, which is necessary for proper muscle function, and they don’t. Because of this, there is a fatal deterioration of muscles, and much like what happens to affected human baby boys, puppies typically die early in life because of breathing difficulties

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The findings, published in Molecular Therapy showed a way to safely replace the MTM-causing defective gene with a healthy gene throughout an affected dog’s entire musculature. Dogs affected with the disease and treated with a single infusion of the gene replacement showed no distinguishable differences when compared to non-affected dogs a year later.

Researcher and physician Dr. Martin K. Childers of the University of Washington said that this treatment therapy and technology gave a complete restoration of normal health to dogs that would typically have died from the disease without it. He also said that the findings were dramatic in result and rescue and give researchers in other inherited disease fields hope for the potential application elsewhere, specifically with relationship to human studies.

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This means that not only is there new hope for affected dogs, but for affected humans, and once again proves dogs to be the leaders in protecting their best friends.