Children with the most common form of muscular dystrophy have been offered hope of living a healthy life after a breakthrough gene therapy was shown to restore muscle strength in dogs.
The hereditary condition, which is marked by progressive weakening and wasting of the muscles, affects one in 5,000 boys and most will not live past their 30s.
However a new trial carried out by researchers at the Royal Holloway in London and French scientists, showed that repairing a defective gene vastly improved the ability of dogs to run, walk and jump.
In some cases, the dogs’ motor skills were indistinguishable from animals without the disease.
Golden Labradors are naturally affected by Duchenne muscular dystrophy so researchers chose them for mammalian trials, ahead of tests in humans.
The 12 dogs, who were not expected to live longer than six months, were treated as puppies and are still alive two years after the trial.
The new therapy works by replacing a faulty gene with fully functioning DNA using a harmless virus, which ‘infects’ cells and alters their genetic code. Once repaired, the gene produces a protein which is essential for correct muscle functioning.
Professor George Dickson, who led the trial at Royal Holloway, said: “This is tremendously exciting progress towards a gene therapy for Duchenne muscular dystrophy.
“The studies in dogs have been spectacular and exceeded our expectations.”
Duchenne muscular dystrophy is the most common type of muscular dystrophy, with 100 boys born with the condition in Britain each year. There are about 2,500 boys living with the disease in the UK at any one time.
Experts said the results offered ‘real hope’ for boys born with the condition.
Prof Darren Griffin, Professor of Genetics, University of Kent, said: “This is really a very exciting study indeed. Duchenne muscular dystrophy is a horrible, wasting, life-ending disease of young people.
“By making use of the canine model and showing genuine improvement in the animals treated, then real hope is present for the prospect for disease treatment in humans.
“The disease has long been a target for gene therapy and it is only to be hoped that sufficient funds can be awarded for this research to reach its natural conclusion and go into full clinical trials.”
The research was published in the journal Nature Communications.