The disease usually caused early death in dogs due to their difficulty breathing.
Gene therapy could be the cure for restoring muscle strength in dogs, which could then lead to a longer life, according to recent reports. Work that is currently being conducted on gene therapy is showing significant progress for strengthening the muscle in dogs who are suffering from a previously incurable, inherited neuromuscular disease which also affects their growth. The disease is caused by a mutation in certain genes that normally produce a protein called myotubularin, which is essential for proper muscle function in dogs. Symptoms of this naturally occurring disease are usually visible when the dogs are young puppies, and they normally exhibit several of the features that babies with the same defective gene also demonstrate. The disorder, which only affects males, is relatively rare, and is called myotubular myopathy, or MTM. The major result of the disease is that it causes fatal muscle wasting, meaning that both dogs and boys who suffer from the disease will typically succumb in their early life due to severe breathing difficulties.
For the past few decades, researchers have been working to find a suitable treatment for the genetic muscle disease, and others similar, but this has previously been to no avail. However, recent reports have now claimed that four collaborating research groups, in the United States and France, have now found a way to safely replace the gene that causes MTM with a healthy gene, throughout the entire musculature of dogs that are affected by it. The scientific paper reports that the diseased dogs that had been treated with just one single infusion of the corrective therapy were then indistinguishable from normal animals who had never had the disease, within the space of just one year. Dr. Martin K. “Casey” Childers, a UW Medicine researcher and physician, describes the success of the new research by saying, “This regenerative technology allowed dogs that otherwise would have perished to complete restoration of normal health.”
The newly discovered and developed gene therapy holds the promise to successfully treat a number of inherited diseases, alongside MTM. So far, this new approach has not been widely translated into the treatment of skeletal muscle disorders, but the research is ongoing. Dr Childers said, “We report here a gene therapy dose-finding study in a large animal model of a severe muscle disease where a single treatment resulted in dramatic rescue.” The scientific findings have therefore demonstrated that there is now a potential to apply the same principles and applications to a wide range of diseases, and even broaden the spectrum to similar research and application to human sufferers of such diseases. The researchers then concluded that their data supports the development of gene therapy clinical trials for myotubular myopathy, making the study a huge success.
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