Duchenne muscular dystrophy (DMD) is the most common and, sadly, the most severe form of the debilitating genetic disorder. Now researchers have used a new gene therapy technique to restore muscle strength and stabilize the symptoms of the disorder in dogs, in an important step that could one day lead to the treatment being applied to children.
[DMD] disrupts dystrophin, a protein that’s responsible for maintaining muscle integrity and strength.
Researchers from the Généthon laboratory at AFM-Téléthon and the Royal Holloway University of London developed microdystrophin, a shortened version of the dystrophin gene that contains only about 4,000 base pairs. When this is combined with a viral vector and injected into a patient, functional proteins are once again produced.
The scientists tested their new gene therapy technique on 12 Golden Retrievers that were naturally affected by DMD. They injected microdystrophin into the animals intravenously, and observed them for more than two years afterwards. After just one dose, the researchers noted that dystrophin production returned to normal, significantly restoring muscle function in the dogs and stabilizing their other symptoms without any side effects.
[The original study can be found here]
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