Gene therapy offers hope for muscular dystrophy sufferers

CHILDREN with the most common form of muscular dystrophy have been offered hope of living a healthy life after a gene therapy was shown to restore muscle strength in dogs.

The hereditary condition, marked by progressive weakening and wasting of the muscles, affects one in 5,000 boys. Most will not live past their 30s.

A trial carried out by researchers at the Royal Holloway in London and in France showed that repairing a defective gene vastly improved the ability of dogs to run, walk and jump. In some cases, their motor skills were indistinguishable from animals without the disease. Golden Labradors are naturally affected by Duchenne muscular dystrophy, so researchers chose them for the trials. The 12 dogs, which were not expected to live longer than six months, were treated as puppies and are still alive two years after the trial.

The therapy works by replacing a faulty gene with fully functioning DNA using a harmless virus, which “infects” cells and alters their genetic code.

The research was published in the journal Nature Communications.