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A Huge Step In Gene Editing Towards Treatment Of Deadly Muscle Disease

Duchenne Muscular Dystrophy [DMD] is a condition, which leads to loss of muscle function and strength and eventually an early death and has no cure. For the first time, scientists have used gene-editing to treat DMD in a large mammal, a remarkable step towards effective treatment for people with the disorder.

Scientists were able to partly restore the main protein people with DMD cannot make in a study carried out on dogs. They hope the technique will work on people in the future.

According to BBC, Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease in children and almost entirely affects boys and young men – about 2,500 of them in the UK have the condition.

Children born with the degenerative disease have a genetic mutation that stops them from producing dystrophin, a protein that is vital for muscle strength and function. This condition also occurs in dogs.

Scientists were able to restore dystrophin in four dogs that had the most common genetic mutation seen in DMD patients, using the CRISPR gene-editing tool, by making a single strategic cut in the faulty DNA.

One-month-old dogs were injected with two safe viruses that edited the genome of the dog in the cells of the muscles and heart.

According to the study in the journal Science. within some weeks, the missing protein was restored in muscle tissue throughout the body, including a 92% correction in the heart and 58% in the diaphragm, the main muscle needed for breathing.

Richard Piercy, professor of comparative neuromuscular disease at the Royal Veterinary College, said: “The ambition is to show that this is safe and effective in dogs and then move into humans trials.

“If that works, then the treatment could also apply to pet dogs that we see in our clinics – and that’s what we hope for here at the college, as it’s our goal to make animals better.”

Some experts have seen the promise in the study but are concerned that only a small group of breed of dogs were used in the study.

Although this is not really a cure for DMD, but it is a step to prove that CRISPR technology could work for DMD.

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