An amazing new breakthrough in gene therapy gives hope for people with muscular dystrophy after researchers reversed the disease in dogs.
Muscular dystrophy has a devastating impact on sufferers, as musculature weakens and degenerates until the patient loses most of their mobility. The symptoms include general muscle weakness and degeneration, stiff joints, coordination and mobility troubles, and frequent falls.
The exciting news for patients is that researchers have been able to reverse the disease in dogs, thanks to new breakthroughs in gene therapy.
What is gene therapy?
Gene therapy is used to replace missing or defective genes in the DNA of a given cell, and has become increasingly more sophisticated over the years. Safe and effective, it has created exciting new pathways in the management of many chronic diseases. Gene editing can be used to treat pathologies, and scientists are also in the early stages of learning how to prevent them in the first place.
Research team reverses muscular dystrophy in dogs
One recent experiment has given people with muscular dystrophy reason to hope that a cure might be on the way. An international research team comprised of scientists from France and the UK recently announced they’d managed to treat Duchenne muscular dystrophy (DMD) with gene therapy in dogs. The findings have been published in Nature Communications, with possible human trials to come.
The research team were able to demonstrate the efficacy of gene therapy in restoring normal muscle function in 12 golden retrievers who were affected by canine DMD. How did they do it? The dogs were injected with highly functional micro-dystrophin genes through a drug vector (a harmless virus) so that the repaired gene could produce the protein involved in muscle function.
The end result of the gene therapy treatment was a stabilisation of clinical symptoms, and two years after the injection of the drug, researchers observed that all dogs showed a significant restoration of their muscles, in addition to regaining a number motor skill. To put this in perspective, none of the dogs were even expected to live beyond the six-month mark.
The great news for muscular dystrophy patients is that this research opens the door to possible human trials in the very near future, with great potential for scientists to discover ways to alleviate – or even cure – their disease.