infographic on gene therapy for dog muscle wasting disease Credit: Martin Childer lab/UW Medicine
In a study replacing the mutated gene responsible for myotubular myopathy with a healthy gene throughout the entire musculature of affected dogs, researchers observed a relationship between dosage and survival.

Gene therapy treats muscle wasting disease in dogs

Single infusion restores strength and prolongs lives of affected canines in latest study.

Work on gene therapy is showing significant progress for restoring muscle strength and prolonging the lives of dogs with a previously incurable, inherited neuromuscular disease. University of Washington Medicine Institute for Stem Cell & Regenerative Medicine scientists are leading the multi-institutional research effort.

The disease arises from a mutation in genes that normally make a protein called myotubularin that is essential for proper muscle function. Puppies with this natu

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