There is new hope for muscular dystrophy patients as researchers, using gene therapy, successfully reversed the disease in dogs.
Gene therapy aims to replace missing or defective genes in the DNA of a given cell.
The technique has evolved over the years to become a viable therapy that’s safe and effective, opening new paths in the management of many difficult diseases.
Not only can gene editing be used to treat pathologies, but it can also to prevent them. Only, we’re not there just yet.Successful muscular dystrophy treatment on animals means humans are next.Click To Tweet
Muscular Dystrophy, one of the Most Debilitating Diseases
Muscular dystrophy is the term used for a group of diseases in which musculature weakens and progressively degenerates until the patient loses most, if not all of their mobility.
Symptoms of muscular dystrophy often include general muscle weakness and degeneration, stiff joints, coordination and mobility troubles, and frequent falls.
In most cases a congenital condition, muscular dystrophy disorders are rare. Each disorder of muscular dystrophy is associated with distinct genetic mutations. The nature and location of the genetic mutation define the form of muscular dystrophy.
Although they can occur at any age, the onset of most MD disorders starts during childhood, and usually, affected persons don’t live past 30 years of age, especially with particularly aggressive forms of the disease like Duchenne muscular dystrophy.
The most common and most studied form of muscular dystrophy is Duchenne muscular dystrophy (DMD), which affects 1 in 5,000 children at birth, and especially boys (1/3500).
After Effective Treatment, Gene Therapy Could Cure Muscular Dystrophy
There’s hope for children and other DMD patients, as a scientific experiment suggests that the disease could be reversed and a cure might be on the way.
An international research team, comprised of scientists from Genethon and Inserm in France and the Royal Holloway at the University of London UK, announced they’d managed to treat Duchenne muscular dystrophy (DMD) with gene therapy in dogs.
Their findings were published in the journal Nature Communications.
The team has shown the efficacy of gene therapy in restoring normal muscle function in 12 dogs (Golden Retrievers) affected by canine DMD, with a stabilization of clinical symptoms.
A video of these dogs before and after treatment can be found here.
Researchers injected highly functional micro-dystrophin genes (a short version of the dystrophin gene) through a drug vector (harmless virus) so that the repaired gene could produce the protein involved in muscle function.
2 years after the injection of the drug, researchers observed that all dogs demonstrated signs of significant restoration of their muscles and regained their motor skills. Not to mention that the same dogs weren’t expected to live past the age of 6 months.
Now, with the method has been shown to be safe and efficient in animals, the next logical step would human trials.
For the many people affected by this debilitating disease, this is a miraculous development.