Can CRISPR Combat Muscular Problems?

Muscular dystrophy currently affects roughly 70,000 Brits, with symptoms ranging from weakness and pain to severe paralysis and death. In a move that has the medical industry buzzing, a team of researchers have successfully used CRISPR gene editing technology to reverse muscular dystrophy symptoms in dogs. Eventually, the team hope that it could be used to help people with the muscle-wasting disease, which comes in around 60 different forms.

While it's considered a rare disease, around 100 British boys are born with Duchenne MD every year. The inherited genetic condition sees abnormal genes, also known as mutations, hinder with the natural production of dystrophin, a protein needed to grow and support healthy muscles. Mutations are often grouped in a certain region of the gene and can eventually stop protein production altogether. Symptoms begin as early as age three and can quickly escalate, with degeneration often spreading to the heart and respiratory muscles by the early teens. It's rare for patients to live past their 20s, with heart failure usually a common cause of death.

Gene editing technology used to repair puppy DNA

Working with beagle puppies suffering from muscular dystrophy, scientists used CRISPR/Cas9 to manually repair the genetic mutation that hinders the production of dystrophin and triggers muscle degeneration. The technology is powered by two key molecules that forcibly change sequences by binding a scaffold to the DNA, then using a pre-designed sequence to guide Cas9 to the correct part of the genome. The repairs were done by injecting the puppies with a virus carrying the CRISPR gene-editing machinery, then noting the effect on dystrophin levels. After eight weeks they noted that levels of dystrophin increased in every muscle group.

“It was really gratifying to see the efficiency with which dystrophin was restored after only eight weeks,” says Eric Olson, co-author of the study and molecular biologist at the University of Texas Southwestern Medical Centre in Dallas.

Pioneering a cure for muscular dystrophy diseases

The findings were reported in the journal Science and confirm that the medical sphere could be one step closer to developing a treatment for muscular dystrophy diseases. Even small improvements could dramatically improve the lives of patients, with previous studies suggesting that restoring dystrophin to just 15 percent of the levels seen in healthy humans could significantly relieve muscular dystrophy symptoms.

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