Gene Editing Has Successfully Treated Muscular Dystrophy in Dogs – Human Trials Next

DNA

In new research published on Monday, an international team of researchers report that they have been able to reverse the symptoms of Duchenne muscular dystrophy in dogs using a single injection of a novel gene therapy.

The symptoms of the disease did not return in the dogs for the two year duration of the study.

Duchenne muscular dystrophy is a severe from of muscle wasting disease that affects one in 5000 males at birth and the average expectancy of patients is 26 years. The disease is genetic and caused by mutations that disrupt the production of dystrophin, which is required for muscles to function properly.

The team comprised of researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR) and the Royal Holloway University of London said in a statement that these results are “paving the way for human clinical trials.”

The researchers created a novel gene transfer therapy that restores the dystrophin expression. They injected the dogs with with the gene microdystrophin, a compressed version of the dystrophin gene. Researchers had previously been unable to successfully use the dystrophin gene in gene therapy due to its relatively large size of 2.3 million base pairs.

After a single injection the dogs in the study demonstrated “significant restoration of muscle function with a stabilisation of clinical symptoms for more than two years after injection,” said the team.

This is “really a very exciting study indeed”, said Geneticist Darren Griffin of the University of Kent.

“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,” he said, quoted by the AFP.

Muscle weakness caused by Duchenne muscular dystrophy usually appears around the age of four in boys and advances rapidly leaving most unable to walk by the age of 12.

Few patients of Duchenne muscular dystrophy survive into their 40s.

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