Gene therapy treats muscle-wasting disease in dogs.

The muscle wasting disease arises from a mutation in genes that normally make a protein, called myotubularin, essential for proper muscle function. Puppies with this naturally occurring mutation exhibit several features of babies with the same defective gene. The rare disorder, called myotubular myopathy, or MTM, affects only males. Puppies with this naturally occurring, fatal genetic mutation and babies with the same defective gene have several similar symptoms. Four collaborating research groups in the United States and France found a way to safely replace the disease-causing MTM gene with a healthy gene throughout the entire musculature of affected dogs. The findings published online in Molecular Therapy ,reports that diseased dogs treated with a single infusion of the corrective therapy were indistinguishable from normal animals one year later. Gene therapy holds the promise to treat many inherited diseases. To date, this approach has not been widely translated into treatment of skeletal muscle disorders.