Focus on gene therapy is showing significant progress for restoring muscle tissue strength and prolonging lives in dogs with a previously not curable, inherited neuromuscular disease, according to scientists at the University associated with Washington Medicine Institute for Stem Cell and Regenerative Medicine.

The disease arises from a mutation in genes that will normally make myotubularin, a protein essential for proper muscle tissue function. Puppies with this naturally occurring mutation exhibit various features of babies with the same defective gene. The uncommon disorder, called myotubular myopathy, or MTM, affects just males. It causes fatal muscle wasting. Both canines and boys with the disease typically succumb in early existence due to breathing difficulties.  

For decades, researchers have battled to find suitable treatments for genetic muscle diseases like here. Collaborating research groups in the United States and France found a method to safely replace the disease-causing MTM gene with a healthful gene…