Have scientists made a breakthrough in DMD research?

Scientists and researchers all over the globe are working tirelessly to find a way of combating Duchenne muscular dystrophy (DMD) – a condition widely considered to be the most common fatal genetic disorder in children.

Currently, DMD affects as many as one in every 3,500 boys worldwide and the muscle-wasting condition causes severe breathing complications for youngsters over time.

Most symptoms do not appear until a child is between three and five-years-old. At first, patients will experience a weakening of the hips, shoulders and thighs, but shortly afterwards their heart and breathing muscles will weaken, too.

Fortunately, researchers hard at work in the field of gene-editing think that they might have found a way of ‘rebuilding’ damaged muscles that have been affected by the disease.

However, so far, studies have only been carried out on dogs – and it remains to be seen whether the innovative gene-editing technology will have the same effects on humans.

DMD is caused in part by the body failing to produce enough of a protein known as dystrophin – which is essential for effective muscle function.

However, by ‘editing’ damaged dog DNA, researchers have seen “unprecedented” success in being able to reboot the body’s production of the protein.

If this process proves successful in humans it could “hopefully prevent patients from dying of the disease” in the near future, scientists from the University of Texas have said.

Dr Leonela Amoasii, lead author of the study, said: “Our strategy is different from other therapeutic approaches for DMD because it edits the mutation that causes the disease and restores normal expression of the repaired dystrophin. But we have more to do before we can use this clinically.”

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