Gene-editing breakthrough good news for muscular dystrophy research

A breakthrough in the field of gene-editing could provide hope for hundreds of people living with muscular dystrophy, researchers have said.

In recent days, scientists have for the first time been able to treat Duchenne muscular dystrophy (DMD) in dogs using an experimental gene-editing procedure.

The condition, which is becoming increasingly common among human children, and especially young boys, currently has no known cure – therefore it is hoped that this breakthrough will lead to innovative new treatments in the near future.

The research, which was led by scientists in the US, injected 20 trillion viruses – each of which carried the Crispr gene-editing tool – into the lower leg muscles of two four-month old dogs with muscular dystrophy.

The two beagles were then tested six months later – at which point researchers realised that levels of dystrophin in the dogs’ muscle fibres had been restored by as much as 60 per cent.

Published in the journal Science in recent days, the research comes shortly after a separate study indicated that boosting dystrophin levels to approximately 15 per cent of normal levels could have huge benefits for muscular dystrophy patients.

Astonishingly, researchers were also able to restore missing proteins into muscle tissues all throughout the body using just a single strategic cut in the faulty DNA.

They discovered a 92 per cent correction in the heart and 58 per cent in the diaphragm of the dog subjects studied, the report reveals.

Study author Dr Eric Olson, of UT Southwestern, said: “Children with DMD often die either because their heart loses the strength to pump or their diaphragm becomes too weak to breathe.

“This encouraging level of dystrophin expression would hopefully prevent that from happening.

Researchers have said that their next step will be to prove that the procedure is safe and effective in dogs, before promptly moving on to human trials.

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