Gene therapy treats muscle-wasting disease in dogs

Dogs with an inherited muscle-wasting disorder that was treated with a single infusion of corrective gene therapy were indistinguishable from normal animals one year later. Puppies with this naturally occurring, fatal genetic mutation and babies with the same defective gene have several similar symptoms. Scientists found a way to safely replace the disease-causing MTM gene with a healthy gene throughout the entire musculature of affected dogs, and are now trying to determine the most effective dosage and timing.